JP2011516586A5 - - Google Patents

Download PDF

Info

Publication number
JP2011516586A5
JP2011516586A5 JP2011504295A JP2011504295A JP2011516586A5 JP 2011516586 A5 JP2011516586 A5 JP 2011516586A5 JP 2011504295 A JP2011504295 A JP 2011504295A JP 2011504295 A JP2011504295 A JP 2011504295A JP 2011516586 A5 JP2011516586 A5 JP 2011516586A5
Authority
JP
Japan
Prior art keywords
lipid
nucleic acid
mol
particle
peg
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2011504295A
Other languages
English (en)
Japanese (ja)
Other versions
JP5475753B2 (ja
JP2011516586A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/CA2009/000496 external-priority patent/WO2009127060A1/en
Publication of JP2011516586A publication Critical patent/JP2011516586A/ja
Publication of JP2011516586A5 publication Critical patent/JP2011516586A5/ja
Application granted granted Critical
Publication of JP5475753B2 publication Critical patent/JP5475753B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2011504295A 2008-04-15 2009-04-15 核酸送達用の脂質製剤 Active JP5475753B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US4522808P 2008-04-15 2008-04-15
US61/045,228 2008-04-15
PCT/CA2009/000496 WO2009127060A1 (en) 2008-04-15 2009-04-15 Novel lipid formulations for nucleic acid delivery

Publications (3)

Publication Number Publication Date
JP2011516586A JP2011516586A (ja) 2011-05-26
JP2011516586A5 true JP2011516586A5 (enExample) 2013-05-23
JP5475753B2 JP5475753B2 (ja) 2014-04-16

Family

ID=41198741

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2011504295A Active JP5475753B2 (ja) 2008-04-15 2009-04-15 核酸送達用の脂質製剤

Country Status (14)

Country Link
US (11) US8058069B2 (enExample)
EP (1) EP2279254B1 (enExample)
JP (1) JP5475753B2 (enExample)
CN (1) CN102119217B (enExample)
AU (1) AU2009238175C1 (enExample)
CA (1) CA2721333C (enExample)
DK (1) DK2279254T3 (enExample)
ES (1) ES2638448T3 (enExample)
HU (1) HUE034483T2 (enExample)
IL (1) IL208744A (enExample)
NZ (1) NZ588583A (enExample)
PL (1) PL2279254T3 (enExample)
PT (1) PT2279254T (enExample)
WO (1) WO2009127060A1 (enExample)

Families Citing this family (825)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10214983A1 (de) 2002-04-04 2004-04-08 TransMIT Gesellschaft für Technologietransfer mbH Vernebelbare Liposomen und ihre Verwendung zur pulmonalen Applikation von Wirkstoffen
EP1519714B1 (en) * 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
EP2365077B1 (en) 2004-03-12 2013-05-08 Alnylam Pharmaceuticals, Inc. iRNA agents targeting VEGF
EP1828219A4 (en) * 2004-11-17 2008-07-23 Protiva Biotherapeutics Inc ARNSI SILENCE OF APOLIPOPROTEIN B
US9393315B2 (en) 2011-06-08 2016-07-19 Nitto Denko Corporation Compounds for targeting drug delivery and enhancing siRNA activity
EP4174179B1 (en) 2005-08-23 2025-05-07 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
CN101346393B (zh) * 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
KR101320916B1 (ko) 2006-05-11 2013-10-23 알닐람 파마슈티칼스 인코포레이티드 Pcsk9 유전자의 발현을 억제하기 위한 조성물 및 방법
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
JP5697988B2 (ja) * 2007-12-27 2015-04-08 プロチバ バイオセラピューティクス インコーポレイティッド 干渉rnaを使用したポロ様キナーゼ発現のサイレンシング方法
AU2009241591A1 (en) * 2008-01-31 2009-11-05 Alnylam Pharmaceuticals, Inc. Optimized methods for delivery of DSRNA targeting the PCSK9 gene
CN105267233B (zh) * 2008-03-05 2019-07-26 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因表达的组合物和方法
PL2279254T3 (pl) 2008-04-15 2017-11-30 Protiva Biotherapeutics Inc. Nowe preparaty lipidowe do dostarczania kwasów nukleinowych
PL2299953T3 (pl) 2008-07-14 2017-10-31 Polypid Ltd Kompozycja nośnika leku o przedłużonym uwalnianiu
CA2739170A1 (en) 2008-09-25 2010-04-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
WO2010042877A1 (en) 2008-10-09 2010-04-15 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
HUE026604T2 (hu) * 2008-10-20 2016-06-28 Alnylam Pharmaceuticals Inc Transztiretin-expresszió gátlására szolgáló készítmények és eljárások
EP3757090B1 (en) 2008-11-10 2024-06-12 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
AU2009324534B2 (en) 2008-12-10 2015-07-30 Alnylam Pharmaceuticals, Inc. GNAQ targeted dsRNA compositions and methods for inhibiting expression
WO2010076248A1 (en) * 2008-12-31 2010-07-08 Santaris Pharma A/S Use of lna apob antisense oligomers for the treatment of acute coronary syndromes
CA2754043A1 (en) * 2009-03-12 2010-09-16 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
KR101184928B1 (ko) * 2009-05-14 2012-09-20 한국과학기술연구원 작은 올리고뉴클레오티드의 중합 방법 및 상기 중합 방법에 의하여 제조된 고분자 올리고뉴클레오티드의 용도
EA201270019A1 (ru) 2009-06-15 2012-06-29 Элнилэм Фармасьютикалз, Инк. Двуцепочечная рнк, включенная в липидный состав и мишенью которой является ген pcsk9
US9051567B2 (en) 2009-06-15 2015-06-09 Tekmira Pharmaceuticals Corporation Methods for increasing efficacy of lipid formulated siRNA
DE102009031274A1 (de) 2009-06-30 2011-01-13 Justus-Liebig-Universität Giessen Liposomen zur pulmonalen Applikation
JP5766188B2 (ja) 2009-07-01 2015-08-19 プロチバ バイオセラピューティクス インコーポレイティッド 固形腫瘍に治療剤を送達するための脂質製剤
WO2011000108A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein b
CN105126179B (zh) 2009-07-14 2018-09-25 波利皮得有限公司 持续释放药物载体组合物
US8716464B2 (en) 2009-07-20 2014-05-06 Thomas W. Geisbert Compositions and methods for silencing Ebola virus gene expression
EP2464336A4 (en) 2009-08-14 2013-11-20 Alnylam Pharmaceuticals Inc COMPOSITIONS FORMULATED IN LIPIDS AND METHODS OF INHIBITING THE EXPRESSION OF EBOLA VIRUS GENE
US9181295B2 (en) 2009-08-20 2015-11-10 Sirna Therapeutics, Inc. Cationic lipids with various head groups for oligonucleotide delivery
CN107519133A (zh) 2009-09-15 2017-12-29 阿尔尼拉姆医药品有限公司 脂质配制的组合物及抑制Eg5和VEGF基因的表达的方法
US9187746B2 (en) 2009-09-22 2015-11-17 Alnylam Pharmaceuticals, Inc. Dual targeting siRNA agents
CA2775092A1 (en) 2009-09-23 2011-03-31 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes expressed in cancer
EP2496238A4 (en) 2009-11-03 2013-10-02 Alnylam Pharmaceuticals Inc FORMULATED LIPID COMPOSITIONS AND METHODS FOR INHIBITING TRANSTHYRETIN EXPRESSION (TTR)
EP2496700B1 (en) 2009-11-04 2017-03-01 The University Of British Columbia Nucleic acid-containing lipid particles and related methods
CA2782676C (en) 2009-12-01 2021-06-15 Shire Human Genetic Therapies, Inc. Pharmaceutical compositions for intracellular delivery of nucleic acids and proteins and uses thereof for treatment of ureca cycle metabolic disorders
US8795726B2 (en) 2010-01-19 2014-08-05 Polypid Ltd. Sustained-release nucleic acid matrix compositions
EP4385568A3 (en) 2010-04-06 2025-02-12 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of cd274/pd-l1 gene
EP2558074B1 (en) 2010-04-08 2018-06-06 The Trustees of Princeton University Preparation of lipid nanoparticles
US20130156845A1 (en) * 2010-04-29 2013-06-20 Alnylam Pharmaceuticals, Inc. Lipid formulated single stranded rna
WO2011141705A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
WO2011141703A1 (en) * 2010-05-12 2011-11-17 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
JP2013531634A (ja) 2010-05-24 2013-08-08 メルク・シャープ・エンド・ドーム・コーポレイション オリゴヌクレオチド送達のための新規なアミノアルコールカチオン性脂質
CA3102008A1 (en) 2010-06-02 2011-12-08 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
CN103327963A (zh) * 2010-07-06 2013-09-25 诺华股份有限公司 阳离子水包油乳液
PL2591114T3 (pl) 2010-07-06 2017-08-31 Glaxosmithkline Biologicals Sa Immunizacja dużych ssaków małymi dawkami rna
AU2011276234B2 (en) 2010-07-06 2016-02-25 Glaxosmithkline Biologicals S.A. Liposomes with lipids having an advantageous pKa- value for RNA delivery
PT3243526T (pt) 2010-07-06 2020-03-04 Glaxosmithkline Biologicals Sa Distribuição de arn para despoletar múltiplas vias imunitárias
FI4005592T3 (fi) 2010-07-06 2023-01-13 Virionin kaltaisia kuljetuspartikkeleita itsereplikoituville rna-molekyyleille
US8822663B2 (en) 2010-08-06 2014-09-02 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
LT2606134T (lt) 2010-08-17 2019-07-25 Sirna Therapeutics, Inc. Hepatito b viruso (hbv) geno raiškos slopinimas, tarpininkaujant rnr interferencijai naudojant mažą interferuojančią nukleorūgštį (sina)
US9233997B2 (en) 2010-08-26 2016-01-12 Sirna Therapeutics, Inc. RNA interference mediated inhibition of prolyl hydroxylase domain 2 (PHD2) gene expression using short interfering nucleic acid (siNA)
PT3981427T (pt) 2010-08-31 2022-06-27 Glaxosmithkline Biologicals Sa Lipossomas peguilados para entrega de arn codificador de imunogénio
PL4008357T3 (pl) 2010-08-31 2023-03-06 Glaxosmithkline Biologicals Sa Małe liposomy do dostarczania rna kodującego immunogen
EP3406730B1 (en) 2010-08-31 2022-02-23 Sirna Therapeutics, Inc. Novel single chemical entities and methods for delivery of oligonucleotides
KR101223483B1 (ko) * 2010-09-10 2013-01-17 한국과학기술연구원 전하결합 및 생분해성 공유결합으로 동시에 연결된 고분자―siRNA 나노입자 전달체
JP5961170B2 (ja) 2010-09-20 2016-08-02 サーナ・セラピューティクス・インコーポレイテッドSirna Therapeutics,Inc. オリゴヌクレオチドの送達のための新規な低分子量カチオン性脂質
JP2013545723A (ja) 2010-09-30 2013-12-26 メルク・シャープ・エンド・ドーム・コーポレイション オリゴヌクレオチドの送達のための低分子量カチオン性脂質
RS63430B1 (sr) 2010-10-01 2022-08-31 Modernatx Inc Ribonukleinske kiseline koje sadrže n1-metil-pseudouracile i njihove primene
TR201903651T4 (tr) 2010-10-11 2019-04-22 Glaxosmithkline Biologicals Sa Antijen uygulama platformları.
US9029590B2 (en) 2010-10-21 2015-05-12 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
ES2663009T3 (es) 2010-10-29 2018-04-10 Sirna Therapeutics, Inc. Inhibición de la expresión génica mediada por interferencia por ARN utilizando ácidos nucleicos de interferencia cortos (ANic)
US9067882B2 (en) 2010-11-05 2015-06-30 Sirna Therapeutics, Inc. Low molecular weight cyclic amine containing cationic lipids for oligonucleotide delivery
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
EP2648763A4 (en) 2010-12-10 2014-05-14 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR EXPRESSION INHIBITION OF GENES KLF-1 AND BCL11A
US9193973B2 (en) 2010-12-10 2015-11-24 Alynylam Pharmaceuticals, Inc. Compositions and methods for increasing erythropoietin (EPO) production
SG10201602369PA (en) 2011-03-29 2016-05-30 Alnylam Pharmaceuticals Inc Double-Stranded RNA (dsRNA) For Inhibiting Expression Of TMPRSS6
EP2691101A2 (en) 2011-03-31 2014-02-05 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US20120301537A1 (en) * 2011-05-23 2012-11-29 Delta-Fly Pharma, Inc. LIPOSOME CONTAINING shRNA MOLECULE TARGETING A THYMIDYLATE SYNTHASE AND USE THEREOF
US8592572B2 (en) * 2011-05-23 2013-11-26 Delta-Fly Pharma, Inc. Liposome containing shRNA molecule targeting a thymidylate synthase and use thereof
US9011903B2 (en) 2011-06-08 2015-04-21 Nitto Denko Corporation Cationic lipids for therapeutic agent delivery formulations
US10196637B2 (en) 2011-06-08 2019-02-05 Nitto Denko Corporation Retinoid-lipid drug carrier
CA3107288A1 (en) 2011-06-08 2012-12-13 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mrna delivery
EP2723865B1 (en) 2011-06-21 2019-03-27 Alnylam Pharmaceuticals, Inc. METHODS FOR DETERMINING ACTIVITY OF RNAi IN A SUBJECT
KR20230107908A (ko) 2011-06-21 2023-07-18 알닐람 파마슈티칼스 인코포레이티드 안지오포이에틴-유사 3(ANGPTL3) iRNA 조성물 및 그사용 방법
EP3388068A1 (en) 2011-06-21 2018-10-17 Alnylam Pharmaceuticals, Inc. Composition and methods for inhibition of expression of protein c (proc) genes
MX344807B (es) 2011-06-21 2017-01-09 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibicion de genes para alipoproteina c-iii (apoc3).
WO2012178033A2 (en) 2011-06-23 2012-12-27 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
EP2729165B1 (en) 2011-07-06 2017-11-08 GlaxoSmithKline Biologicals SA Immunogenic combination compositions and uses thereof
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
DK3682905T3 (da) 2011-10-03 2022-02-28 Modernatx Inc Modificerede nukleosider, nukleotider og nukleinsyrer og anvendelser deraf
AU2012318249B2 (en) * 2011-10-05 2016-04-21 Protiva Biotherapeutics Inc. Compositions and methods for silencing aldehyde dehydrogenase
WO2013059922A1 (en) 2011-10-25 2013-05-02 The University Of British Columbia Limit size lipid nanoparticles and related methods
US9320814B2 (en) * 2011-11-01 2016-04-26 Board Of Regents Of The University Of Nebraska Polyplexes of hydrophobically-modified siRNA for delivery of siRNA
MX391815B (es) 2011-11-18 2025-03-21 Alnylam Pharmaceuticals Inc AGENTES DE iARN, COMPOSICIONES Y METODOS DE LOS MISMOS PARA USARSE EN EL TRATAMIENTO DE ENFERMEDADES ASOCIADAS CON TRANSTITERINA (TTR).
CA2856742A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
WO2013086373A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
DE12858350T1 (de) 2011-12-16 2021-10-07 Modernatx, Inc. Modifizierte mrna zusammensetzungen
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
EP3473611B1 (en) * 2012-02-24 2021-10-20 Arbutus Biopharma Corporation Trialkyl cationic lipids and methods of use thereof
US9546128B2 (en) 2012-03-29 2017-01-17 Shire Human Genetic Therapies, Inc. Ionizable cationic lipids
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
CN104411338A (zh) * 2012-04-02 2015-03-11 现代治疗公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
HK1206601A1 (en) 2012-04-02 2016-01-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
US9878056B2 (en) 2012-04-02 2018-01-30 Modernatx, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
US9402816B2 (en) 2012-04-19 2016-08-02 Sima Therapeutics, Inc. Diester and triester based low molecular weight, biodegradeable cationic lipids for oligonucleotide delivery
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
JP6561378B2 (ja) 2012-06-08 2019-08-21 トランスレイト バイオ, インコーポレイテッド 非肺標的細胞へのmRNAの経肺送達
EP2859102A4 (en) 2012-06-08 2016-05-11 Shire Human Genetic Therapies NUCLEASE RESISTANT POLYNUCLEOTIDES AND USES THEREOF
WO2014008334A1 (en) 2012-07-06 2014-01-09 Alnylam Pharmaceuticals, Inc. Stable non-aggregating nucleic acid lipid particle formulations
CN102719437A (zh) * 2012-07-11 2012-10-10 潍坊医学院 利用慢病毒载体介导PLK1 RNAi在治疗食管鳞癌转移中的应用
EP2885419A4 (en) 2012-08-14 2016-05-25 Moderna Therapeutics Inc ENZYMES AND POLYMERASES FOR RNA SYNTHESIS
HRP20220607T1 (hr) 2012-11-26 2022-06-24 Modernatx, Inc. Terminalno modificirana rna
WO2014089313A1 (en) 2012-12-05 2014-06-12 Alnylam Pharmaceuticals PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4299741A3 (en) 2012-12-12 2024-02-28 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
EA201591229A1 (ru) 2013-03-14 2016-01-29 Шир Хьюман Дженетик Терапис, Инк. Способы очистки матричной рнк
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
MX365409B (es) 2013-03-14 2019-05-31 Shire Human Genetic Therapies Composiciones de ácido ribonucleico mensajero del regulador transmembrana de fibrosis quística y métodos y usos relacionados.
RS62565B1 (sr) 2013-03-14 2021-12-31 Translate Bio Inc Metode i kompozicije za isporuku antitela kodiranih od strane irnk
WO2014160129A2 (en) 2013-03-14 2014-10-02 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2014160243A1 (en) 2013-03-14 2014-10-02 The Trustees Of The University Of Pennsylvania Purification and purity assessment of rna molecules synthesized with modified nucleosides
US10077439B2 (en) 2013-03-15 2018-09-18 Modernatx, Inc. Removal of DNA fragments in mRNA production process
EP2983804A4 (en) 2013-03-15 2017-03-01 Moderna Therapeutics, Inc. Ion exchange purification of mrna
WO2014140856A2 (en) 2013-03-15 2014-09-18 Graham Lord Mir-142 and antagonists thereof for treating disease
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
HUE071526T2 (hu) 2013-03-15 2025-09-28 Translate Bio Inc Nukleinsavak bejuttatásának szinergista fokozása kevert kiszerelésekkel
US11377470B2 (en) 2013-03-15 2022-07-05 Modernatx, Inc. Ribonucleic acid purification
EP2971013B1 (en) 2013-03-15 2020-08-19 The University Of British Columbia Lipid nanoparticles for transfection and related methods
EP2971033B8 (en) 2013-03-15 2019-07-10 ModernaTX, Inc. Manufacturing methods for production of rna transcripts
KR102365809B1 (ko) 2013-05-22 2022-02-23 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 조성물 및 이의 사용 방법
CA2912826A1 (en) 2013-05-22 2014-11-27 Alnylam Pharmaceuticals, Inc. Serpina1 irna compositions and methods of use thereof
BR112015031608A2 (pt) 2013-06-17 2017-08-22 Massachusetts Inst Technology Aplicação e uso dos sistemas crispr-cas, vetores e composições para direcionamento e terapia hepáticos
ES2767318T3 (es) 2013-06-17 2020-06-17 Broad Inst Inc Suministro, modificación y optimización de sistemas, métodos y composiciones para generar modelos y actuar sobre enfermedades y trastornos de células posmitóticas
BR122021009076B1 (pt) 2013-06-17 2024-02-15 The Broad Institute Inc. Vetor viral contendo molécula(s) de ácido nucleico heterólogo, composição, uso e métodos do mesmo
RS62529B1 (sr) 2013-07-11 2021-11-30 Modernatx Inc Kompozicije koje sadrže sintetičke polinukleotide koji kodiraju proteine povezane sa crispr i sintetičke sgrnk i postupci upotrebe
US20160151284A1 (en) * 2013-07-23 2016-06-02 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
WO2015034925A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Circular polynucleotides
US20160194625A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
WO2015051169A2 (en) 2013-10-02 2015-04-09 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
TW202003849A (zh) 2013-10-02 2020-01-16 美商艾爾妮蘭製藥公司 抑制lect2基因表現之組合物及方法
WO2015051214A1 (en) 2013-10-03 2015-04-09 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
PE20161130A1 (es) 2013-10-04 2016-11-25 Icahn School Med Mount Sinai Composiciones y metodos para inhibir la expresion del gen alas1
CA2928040A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Cns delivery of mrna and uses thereof
EP3871696B1 (en) 2013-10-22 2025-03-19 Translate Bio, Inc. Lipid formulations for delivery of messenger rna
MX2016005239A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Tratamiento con acido ribonucleico mensajero para la fenilcetonuria.
EP3060303B1 (en) 2013-10-22 2018-11-14 Translate Bio, Inc. Mrna therapy for argininosuccinate synthetase deficiency
CN112220797B (zh) 2013-11-22 2023-11-03 米纳治疗有限公司 C/EBPα组合物和使用方法
EP3653703A1 (en) 2013-12-12 2020-05-20 The Broad Institute, Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
WO2015089462A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
SG10201804975PA (en) 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for HBV and Viral Diseases and Disorders
SG10201804977UA (en) 2013-12-12 2018-07-30 Broad Inst Inc Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for Targeting Disorders and Diseases Using Particle Delivery Components
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
MX382900B (es) 2013-12-12 2025-03-13 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ÁCIDO RIBONUCLEICO DE INTERFERENCIA (ARNi) CONTRA EL COMPONENTE DEL COMPLEMENTO Y MÉTODOS PARA SU USO.
EP3960860A3 (en) 2014-02-11 2022-06-08 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
EP3556353A3 (en) 2014-02-25 2020-03-18 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
DK3134131T3 (en) 2014-04-23 2022-02-07 Modernatx Inc Nucleic acid vaccines
KR20250005450A (ko) 2014-04-25 2025-01-09 샤이어 휴먼 지네틱 테라피즈 인크. 메신저 rna 의 정제 방법
EA201692370A1 (ru) 2014-05-22 2017-03-31 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ иРНК АНГИОТЕНЗИНОГЕНА (AGT) И СПОСОБЫ ИХ ИСПОЛЬЗОВАНИЯ
BR112016027705A2 (pt) 2014-05-30 2018-01-30 Shire Human Genetic Therapies lipídios biodegradáveis para distribuição de ácidos nucleicos
EP3154694B1 (en) 2014-06-13 2025-01-29 Children's Medical Center Corporation Products and methods to isolate mitochondria
US10286086B2 (en) 2014-06-19 2019-05-14 Modernatx, Inc. Alternative nucleic acid molecules and uses thereof
PE20171238A1 (es) 2014-06-24 2017-08-24 Shire Human Genetic Therapies Composiciones enriquecidas estereoquimicamente para administracion de acidos nucleicos
PT3766916T (pt) 2014-06-25 2022-11-28 Acuitas Therapeutics Inc Novos lípidos e formulações de nanopartículas lipídicas para distribuição de ácidos nucleicos
CN114146063A (zh) 2014-07-02 2022-03-08 川斯勒佰尔公司 信使rna的包封
AU2015289583A1 (en) 2014-07-16 2017-02-02 Modernatx, Inc. Chimeric polynucleotides
CN106794141B (zh) * 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
EP3169335B8 (en) 2014-07-16 2019-10-09 ModernaTX, Inc. Circular polynucleotides
EP3171895A1 (en) 2014-07-23 2017-05-31 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
DK3180426T3 (da) 2014-08-17 2020-03-30 Broad Inst Inc Genomredigering ved anvendelse af cas9-nickaser
US9765333B2 (en) 2014-08-21 2017-09-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing marburg virus gene expression
WO2016033326A2 (en) 2014-08-29 2016-03-03 Alnylam Pharmaceuticals, Inc. Methods of treating transthyretin (ttr) mediated amyloidosis
EP3191591A1 (en) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
CA2963271A1 (en) 2014-10-02 2016-04-07 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis b virus gene expression
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
WO2016061487A1 (en) 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3904519A1 (en) 2014-10-30 2021-11-03 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
CA2968114A1 (en) 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
WO2016090262A1 (en) 2014-12-05 2016-06-09 Shire Human Genetic Therapies, Inc. Messenger rna therapy for treatment of articular disease
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
CA2970795A1 (en) 2014-12-18 2016-06-23 Alnylam Pharmaceuticals, Inc. Reversir compounds
EP3234192B1 (en) 2014-12-19 2021-07-14 The Broad Institute, Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Crispr having or associated with destabilization domains
WO2016118697A1 (en) 2015-01-21 2016-07-28 Phaserx, Inc. Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
JP2018510621A (ja) 2015-02-13 2018-04-19 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. パタチン様ホスホリパーゼドメイン含有3(PNPLA3)iRNA組成物およびその使用方法
JP6895892B2 (ja) 2015-03-19 2021-06-30 トランスレイト バイオ, インコーポレイテッド ポンペ病のmRNA治療
US10745702B2 (en) 2015-04-08 2020-08-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
EP3283059B1 (en) 2015-04-13 2024-01-03 CureVac Manufacturing GmbH Method for producing rna compositions
CN104997801A (zh) * 2015-05-28 2015-10-28 厦门成坤生物技术有限公司 包含季铵盐类化合物和siRNA的组合物及其制备方法
US20180245074A1 (en) 2015-06-04 2018-08-30 Protiva Biotherapeutics, Inc. Treating hepatitis b virus infection using crispr
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
JP7107683B2 (ja) 2015-06-18 2022-07-27 ザ・ブロード・インスティテュート・インコーポレイテッド オフターゲット効果を低下させるcrispr酵素突然変異
EP3310918B1 (en) 2015-06-18 2020-08-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016205745A2 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
CA3012607A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzymes and systems
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
AU2016285852B2 (en) 2015-06-29 2020-12-17 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
US10494632B2 (en) 2015-07-10 2019-12-03 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (IGFALS) compositions and methods of use thereof
KR20180027532A (ko) * 2015-07-10 2018-03-14 펩티노보 바이오파마, 엘엘씨 소수성 약물의 효능 개선용 제형
WO2017015463A2 (en) 2015-07-21 2017-01-26 Modernatx, Inc. Infectious disease vaccines
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
HK1248278A1 (zh) 2015-07-29 2018-10-12 Arbutus Biopharma Corporation 用於使乙型肝炎病毒基因表达沉默的组合物及方法
US12150980B2 (en) 2015-07-30 2024-11-26 Modernatx, Inc. Concatemeric peptide epitope RNAs
KR20240074895A (ko) 2015-07-31 2024-05-28 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴(TTR) iRNA 조성물 및 TTR-관련 질병을 치료하거나, 예방하기 위한 그의 사용 방법
US11564893B2 (en) 2015-08-17 2023-01-31 Modernatx, Inc. Methods for preparing particles and related compositions
AU2016308339A1 (en) 2015-08-18 2018-04-12 Baylor College Of Medicine Methods and compositions for altering function and structure of chromatin loops and/or domains
WO2017035278A1 (en) 2015-08-24 2017-03-02 Halo-Bio Rnai Therapeutics, Inc. Polynucleotide nanoparticles for the modulation of gene expression and uses thereof
CA2996873A1 (en) 2015-09-02 2017-03-09 Alnylam Pharmaceuticals, Inc. Programmed cell death 1 ligand 1 (pd-l1) irna compositions and methods of use thereof
EP3350328A1 (en) 2015-09-14 2018-07-25 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof
US11434486B2 (en) 2015-09-17 2022-09-06 Modernatx, Inc. Polynucleotides containing a morpholino linker
CA2998370A1 (en) 2015-09-17 2017-03-23 Moderna Therapeutics, Inc. Polynucleotides containing a stabilizing tail region
WO2017049245A2 (en) 2015-09-17 2017-03-23 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
CA3001003A1 (en) 2015-10-05 2017-04-13 Modernatx, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
WO2017069958A2 (en) 2015-10-09 2017-04-27 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
ES2862412T3 (es) 2015-10-14 2021-10-07 Translate Bio Inc Modificación de enzimas relacionados con el ARN para producción mejorada
WO2017066726A1 (en) 2015-10-16 2017-04-20 Merrimack Pharmaceuticals, Inc. Stabilizing camptothecin pharmaceutical compositions
MA45209A (fr) 2015-10-22 2019-04-17 Modernatx Inc Vaccins contre les maladies sexuellement transmissibles
MA46080A (fr) 2015-10-22 2019-07-10 Modernatx Inc Vaccins à base d'acide nucléique contre le virus varicelle-zona (vzv)
US12234454B2 (en) 2015-10-22 2025-02-25 The Broad Institute, Inc. Crispr enzymes and systems
CA3002922A1 (en) * 2015-10-22 2017-04-27 Modernatx, Inc. Human cytomegalovirus vaccine
EP3364950A4 (en) 2015-10-22 2019-10-23 ModernaTX, Inc. VACCINES AGAINST TROPICAL DISEASES
WO2017074788A1 (en) 2015-10-27 2017-05-04 The Broad Institute Inc. Compositions and methods for targeting cancer-specific sequence variations
WO2017075451A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
WO2017075465A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
EP3368689B1 (en) 2015-10-28 2020-06-17 The Broad Institute, Inc. Composition for modulating immune responses by use of immune cell gene signature
LT3368507T (lt) 2015-10-28 2023-03-10 Acuitas Therapeutics Inc. Nauji lipidai ir lipidų nanodalelių kompozicijos, skirtos nukleorūgščių tiekimui
WO2017095946A1 (en) 2015-11-30 2017-06-08 Flagship Pioneering, Inc. Methods and compositions relating to chondrisomes
JP7080172B2 (ja) 2015-12-10 2022-06-03 モデルナティエックス インコーポレイテッド 治療薬の送達のための組成物及び方法
US12110490B2 (en) 2015-12-18 2024-10-08 The Broad Institute, Inc. CRISPR enzymes and systems
ES2913626T5 (en) 2015-12-22 2025-05-12 Modernatx Inc Compounds and compositions for intracellular delivery of agents
SI3394093T1 (sl) 2015-12-23 2022-05-31 Modernatx, Inc. Metode uporabe liganda OX40, ki kodira polinukleotid
WO2017120612A1 (en) 2016-01-10 2017-07-13 Modernatx, Inc. Therapeutic mrnas encoding anti ctla-4 antibodies
EP3402490B1 (en) 2016-01-15 2022-06-01 The Children's Medical Center Corporation Therapeutic use of mitochondria and combined mitochondrial agents
CA3020343A1 (en) 2016-04-08 2017-10-12 Translate Bio, Inc. Multimeric coding nucleic acid and uses thereof
MA45295A (fr) 2016-04-19 2019-02-27 Alnylam Pharmaceuticals Inc Composition d'arni de protéine de liaison de lipoprotéines haute densité (hdlbp/vigiline) et procédés pour les utiliser
EP3445853A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
WO2017189308A1 (en) 2016-04-19 2017-11-02 The Broad Institute Inc. Novel crispr enzymes and systems
KR102424476B1 (ko) 2016-04-19 2022-07-25 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
WO2017201340A2 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding relaxin
JP7194594B2 (ja) 2016-05-18 2022-12-22 モデルナティエックス インコーポレイテッド 免疫調節ポリペプチドをコードするmRNAの組み合わせ及びその使用
JP7246930B2 (ja) * 2016-05-18 2023-03-28 モデルナティエックス インコーポレイテッド インターロイキン-12(il12)をコードするポリヌクレオチドおよびその使用
US12128113B2 (en) 2016-05-18 2024-10-29 Modernatx, Inc. Polynucleotides encoding JAGGED1 for the treatment of Alagille syndrome
US20190256845A1 (en) 2016-06-10 2019-08-22 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
CN109312313A (zh) 2016-06-13 2019-02-05 川斯勒佰尔公司 用于治疗鸟氨酸转氨甲酰酶缺乏症的信使rna疗法
AU2017286606A1 (en) * 2016-06-14 2018-12-13 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
CA3028158A1 (en) 2016-06-17 2017-12-21 The Broad Institute, Inc. Type vi crispr orthologs and systems
US12385034B2 (en) 2016-06-24 2025-08-12 Modernatx, Inc. Methods and apparatus for filtration
US20210222164A1 (en) 2016-06-29 2021-07-22 The Broad Institute, Inc. Crispr-cas systems having destabilization domain
JP7086870B2 (ja) 2016-06-30 2022-06-20 アルブータス・バイオファーマー・コーポレイション メッセンジャーrnaを送達するための組成物及び方法
US20190161730A1 (en) 2016-07-07 2019-05-30 Rubius Therapeutics, Inc. Compositions and methods related to therapeutic cell systems expressing exogenous rna
US20200283743A1 (en) 2016-08-17 2020-09-10 The Broad Institute, Inc. Novel crispr enzymes and systems
EP3500671B1 (en) 2016-08-17 2024-07-10 The Broad Institute, Inc. Method of selecting target sequences for the design of guide rnas
SG11201811432WA (en) 2016-08-19 2019-03-28 Curevac Ag Rna for cancer therapy
EP3500294A4 (en) 2016-08-22 2020-07-29 Arbutus Biopharma Corporation ANTI-PD-1 ANTIBODIES, OR THEIR FRAGMENTS, FOR THE TREATMENT OF HEPATITIS B
US20190262399A1 (en) 2016-09-07 2019-08-29 The Broad Institute, Inc. Compositions and methods for evaluating and modulating immune responses
WO2018064208A1 (en) 2016-09-28 2018-04-05 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
EP3519578B1 (en) 2016-10-03 2021-12-22 Precision Nanosystems Inc Compositions for transfecting resistant cell types
WO2018119514A1 (en) * 2016-12-28 2018-07-05 Precision Nanosystems Inc. Compositions for transfecting resistant cell types
US12447213B2 (en) 2016-10-07 2025-10-21 The Broad Institute, Inc. Modulation of novel immune checkpoint targets
WO2018075980A1 (en) 2016-10-21 2018-04-26 Modernatx, Inc. Human cytomegalovirus vaccine
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
EP3532103B1 (en) 2016-10-26 2025-12-03 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
EP3538067A1 (en) 2016-11-08 2019-09-18 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
EP3538146A4 (en) 2016-11-11 2020-07-15 ModernaTX, Inc. INFLUENZA VACCINE
TWI788312B (zh) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子A1 iRNA組成物及其使用方法
US11542490B2 (en) 2016-12-08 2023-01-03 CureVac SE RNAs for wound healing
MA50335A (fr) 2016-12-08 2020-08-19 Modernatx Inc Vaccins à acide nucléique contre des virus respiratoires
JP2020501545A (ja) 2016-12-08 2020-01-23 キュアバック アーゲー 肝疾患の処置または予防のためのrna
TWI865997B (zh) 2016-12-16 2024-12-11 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白(TTR)iRNA組成物於治療或預防TTR相關疾病之方法
EP3558356A2 (en) 2016-12-23 2019-10-30 CureVac AG Mers coronavirus vaccine
EP3558354A1 (en) 2016-12-23 2019-10-30 CureVac AG Lassa virus vaccine
US11524066B2 (en) 2016-12-23 2022-12-13 CureVac SE Henipavirus vaccine
EA201991747A1 (ru) 2017-02-27 2020-06-04 Транслейт Био, Инк. НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR
WO2018170260A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Respiratory syncytial virus vaccine
WO2018170270A1 (en) * 2017-03-15 2018-09-20 Modernatx, Inc. Varicella zoster virus (vzv) vaccine
WO2018170336A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Lipid nanoparticle formulation
EP3596041B1 (en) 2017-03-15 2022-11-02 ModernaTX, Inc. Compound and compositions for intracellular delivery of therapeutic agents
JP7220154B2 (ja) 2017-03-15 2023-02-09 モデルナティエックス インコーポレイテッド アミノ脂質の結晶形態
US11752206B2 (en) * 2017-03-15 2023-09-12 Modernatx, Inc. Herpes simplex virus vaccine
US11576961B2 (en) 2017-03-15 2023-02-14 Modernatx, Inc. Broad spectrum influenza virus vaccine
EP3596207B1 (en) 2017-03-15 2023-12-20 The Broad Institute, Inc. Novel cas13b orthologues crispr enzymes and systems
US20200030432A1 (en) 2017-03-17 2020-01-30 Modernatx, Inc. Zoonotic disease rna vaccines
JP2020514370A (ja) 2017-03-17 2020-05-21 キュアバック アーゲー 組合せ抗癌療法のためのrnaワクチン及び免疫チェックポイント阻害剤
US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
WO2018172556A1 (en) 2017-03-24 2018-09-27 Curevac Ag Nucleic acids encoding crispr-associated proteins and uses thereof
US11905525B2 (en) 2017-04-05 2024-02-20 Modernatx, Inc. Reduction of elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins
JP7679174B2 (ja) 2017-04-12 2025-05-19 ザ・ブロード・インスティテュート・インコーポレイテッド 新規vi型crisprオルソログ及び系
US11357856B2 (en) 2017-04-13 2022-06-14 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
WO2018191719A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipid delivery of therapeutic agents to adipose tissue
US12350368B2 (en) 2017-04-14 2025-07-08 The Broad Institute, Inc. Delivery of large payloads
CA3059446A1 (en) 2017-04-18 2018-10-25 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (hbv) infection
CA3061612A1 (en) 2017-04-28 2018-11-01 Acuitas Therapeutics, Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
US11591601B2 (en) 2017-05-05 2023-02-28 The Broad Institute, Inc. Methods for identification and modification of lncRNA associated with target genotypes and phenotypes
BR112019023323A2 (pt) 2017-05-08 2020-07-21 Flagship Pioneering Innovations V, Inc. composições para facilitar a fusão de membrana e usos das mesmas
CA3063531A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
US12297436B2 (en) 2017-05-18 2025-05-13 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
JP7284101B2 (ja) 2017-05-31 2023-05-30 ウルトラジェニクス ファーマシューティカル インク. 糖原病iii型のための治療薬
ES3039647T3 (en) 2017-05-31 2025-10-23 Arcturus Therapeutics Inc Synthesis and structure of high potency rna therapeutics
WO2018222925A1 (en) 2017-05-31 2018-12-06 Ultragenyx Pharmaceutical Inc. Therapeutics for phenylketonuria
US20200123203A1 (en) 2017-06-13 2020-04-23 Flagship Pioneering Innovations V, Inc. Compositions comprising curons and uses thereof
US12077501B2 (en) 2017-06-14 2024-09-03 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS
AU2018298422B2 (en) 2017-07-04 2023-04-06 CureVac SE Novel nucleic acid molecules
AU2018301477A1 (en) 2017-07-13 2020-02-27 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (LDHA) iRNA compositions and methods of use thereof
CA3073020A1 (en) 2017-08-16 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
CA3073018A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US11542225B2 (en) 2017-08-17 2023-01-03 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036030A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
EP4328314A3 (en) 2017-08-22 2024-03-20 Tessera Therapeutics, Inc. Lipid nanoparticle methods and compositions for producing engineered erythroid cells
US11602557B2 (en) 2017-08-22 2023-03-14 Cure Vac SE Bunyavirales vaccine
JP7275111B2 (ja) 2017-08-31 2023-05-17 モデルナティエックス インコーポレイテッド 脂質ナノ粒子の生成方法
WO2019048645A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
WO2019048631A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited SMALL HNF4A ACTIVATOR RNA COMPOSITIONS AND METHODS OF USE
MA50253A (fr) 2017-09-14 2020-07-22 Modernatx Inc Vaccins à arn contre le virus zika
AU2018336806B2 (en) 2017-09-19 2025-04-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
KR102338449B1 (ko) 2017-09-21 2021-12-10 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물
US20200255828A1 (en) 2017-10-04 2020-08-13 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
IL321714A (en) 2017-10-19 2025-08-01 CureVac SE Novel artificial nucleic acid molecules
US12227742B2 (en) 2017-10-23 2025-02-18 The Broad Institute, Inc. Nucleic acid modifiers
US11547614B2 (en) 2017-10-31 2023-01-10 The Broad Institute, Inc. Methods and compositions for studying cell evolution
WO2019089828A1 (en) 2017-10-31 2019-05-09 Acuitas Therapeutics, Inc. Lamellar lipid nanoparticles
RU2020117848A (ru) 2017-11-08 2021-12-08 Куревак Аг Адаптиция последовательности phk
EP3710039A4 (en) 2017-11-13 2021-08-04 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR TREATMENT OF CANCER BY TARGETING THE CLEC2D-KLRB1 PATH
US20200385719A1 (en) 2017-11-16 2020-12-10 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
EP3714054A1 (en) 2017-11-20 2020-09-30 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
WO2019115635A1 (en) 2017-12-13 2019-06-20 Curevac Ag Flavivirus vaccine
TW201936201A (zh) 2017-12-14 2019-09-16 美商堅固生物科技公司 基因之非病毒生產及遞送
MX2020006012A (es) 2017-12-18 2020-09-14 Alnylam Pharmaceuticals Inc Composiciones de arni de la caja 1 del grupo de alta movilidad (hmgb1) y métodos de uso de las mismas.
AU2018392716B2 (en) 2017-12-20 2025-03-13 Translate Bio, Inc. Improved composition and methods for treatment of ornithine transcarbamylase deficiency
US20230193242A1 (en) 2017-12-22 2023-06-22 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
WO2019131770A1 (ja) * 2017-12-27 2019-07-04 武田薬品工業株式会社 核酸含有脂質ナノ粒子及びその用途
WO2019136432A1 (en) 2018-01-08 2019-07-11 Novartis Ag Immune-enhancing rnas for combination with chimeric antigen receptor therapy
WO2019140116A2 (en) 2018-01-10 2019-07-18 Rubius Therapeutics, Inc. Amplifiable rnas for therapeutic cell systems
EP3746090A4 (en) 2018-01-29 2021-11-17 ModernaTX, Inc. RSV RNA Vaccines
WO2019166877A1 (en) 2018-03-02 2019-09-06 Sixfold Bioscience Ltd. Compositions for delivery of cargo to cells
WO2019193183A2 (en) 2018-04-05 2019-10-10 Curevac Ag Novel yellow fever nucleic acid molecules for vaccination
WO2019197845A1 (en) 2018-04-12 2019-10-17 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
EP4227319B1 (en) 2018-04-17 2025-11-26 CureVac SE Novel rsv rna molecules and compositions for vaccination
CA3098624A1 (en) 2018-05-11 2019-11-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原(AGT)iRNA組成物及其使用方法
JP7178409B2 (ja) 2018-06-08 2022-11-25 富士フイルム株式会社 化合物またはその塩および脂質粒子
CN120393053A (zh) 2018-06-18 2025-08-01 全国儿童医院研究所 用于治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送
US12357580B2 (en) 2018-06-19 2025-07-15 The Board Of Regents Of The University Of Texas System Lipid nanoparticle compositions for delivery of mRNA and long nucleic acids
US20210260178A1 (en) 2018-06-27 2021-08-26 Curevac Ag Novel lassa virus rna molecules and compositions for vaccination
EP3833761A1 (en) 2018-08-07 2021-06-16 The Broad Institute, Inc. Novel cas12b enzymes and systems
EP3837367A1 (en) 2018-08-16 2021-06-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
EP3841208A1 (en) 2018-08-24 2021-06-30 Translate Bio, Inc. Methods for purification of messenger rna
AU2019335055B2 (en) 2018-09-04 2025-05-29 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids
CN112996519B (zh) 2018-09-04 2025-02-28 德克萨斯大学系统董事会 用于核酸的器官特异性递送的组合物和方法
WO2020060986A1 (en) 2018-09-18 2020-03-26 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
EP3853202A1 (en) 2018-09-19 2021-07-28 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
JP7526168B2 (ja) 2018-09-19 2024-07-31 モデルナティエックス インコーポレイテッド Peg脂質及びそれらの使用
US12383508B2 (en) 2018-09-19 2025-08-12 Modernatx, Inc. High-purity peg lipids and uses thereof
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
IL322436A (en) 2018-09-21 2025-09-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
IL281135B2 (en) 2018-09-28 2024-12-01 Nutcracker Therapeutics Inc Tertiary amino lipidated cationic peptides for nucleic acid delivery
CN118307422A (zh) * 2018-10-01 2024-07-09 阿尔尼拉姆医药品有限公司 用于递送活性剂的可生物降解脂质
EA202190688A1 (ru) 2018-10-09 2021-06-07 Дзе Юниверсити Оф Бритиш Коламбиа Композиции и системы, содержащие трансфекционно-компетентные везикулы, не содержащие органических растворителей и детергентов, и связанные с ними способы
US10913951B2 (en) 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
JP2022506956A (ja) 2018-11-09 2022-01-17 アルブータス・バイオファーマー・コーポレイション 脂質ナノ粒子製剤
WO2020097520A1 (en) 2018-11-09 2020-05-14 Arbutus Biopharma Corporation Cationic lipids containing silicon
EP3876999A4 (en) 2018-11-09 2022-08-31 Arbutus Biopharma Corporation NEGATIVELY CHARGED PEG-LIPID CONJUGATES
MX2021005969A (es) 2018-11-21 2021-09-14 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr nebulizado.
US12485136B2 (en) 2018-12-03 2025-12-02 Takeda Pharmaceuticals U.S.A., Inc. Methods for the treatment of trinucleotide repeat expansion disorders associated with MLH3 activity
AU2019394996B2 (en) 2018-12-06 2025-11-06 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
US11166996B2 (en) 2018-12-12 2021-11-09 Flagship Pioneering Innovations V, Inc. Anellovirus compositions and methods of use
TW202039534A (zh) 2018-12-14 2020-11-01 美商美國禮來大藥廠 KRAS變體mRNA分子
US12268669B2 (en) 2018-12-20 2025-04-08 Pfizer Inc. Pharmaceutical compositions and methods comprising a combination of a benzoxazole transthyretin stabilizer and an additional therapeutic agent
US20220056455A1 (en) 2018-12-20 2022-02-24 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of kcnt1 related disorders
EP3897702A2 (en) 2018-12-21 2021-10-27 CureVac AG Rna for malaria vaccines
US12042527B2 (en) 2019-01-08 2024-07-23 Modernatx, Inc. Use of mRNAs encoding OX40L, IL-23 and IL-36gamma in combination with immune checkpoint blockade for treating particular cancers
TWI852977B (zh) 2019-01-10 2024-08-21 美商健生生物科技公司 前列腺新抗原及其用途
WO2020146805A1 (en) 2019-01-11 2020-07-16 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
US20220133908A1 (en) 2019-02-08 2022-05-05 Curevac Ag Coding rna administered into the suprachoroidal space in the treatment of ophthalmic diseases
US11351242B1 (en) 2019-02-12 2022-06-07 Modernatx, Inc. HMPV/hPIV3 mRNA vaccine composition
MA55321A (fr) 2019-03-15 2022-01-19 Modernatx Inc Vaccins à base d'arn contre le vih
WO2020191102A1 (en) 2019-03-18 2020-09-24 The Broad Institute, Inc. Type vii crispr proteins and systems
US20220211740A1 (en) 2019-04-12 2022-07-07 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
US20220220469A1 (en) 2019-05-20 2022-07-14 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
CA3143865A1 (en) 2019-06-07 2020-12-10 Fujifilm Corporation Lipid composition
BR112021024288A2 (pt) 2019-06-07 2022-02-15 Scribe Therapeutics Inc Sistemas casx modificados
US20220313813A1 (en) 2019-06-18 2022-10-06 Curevac Ag Rotavirus mrna vaccine
CA3147875A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
EP4005602A4 (en) 2019-07-30 2024-06-12 Shionogi & Co., Ltd NUCLEIC ACID AGENT TARGETED AGAINST MURF1
WO2021022109A1 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. SERPIN FAMILY F MEMBER 2 (SERPINF2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4007811A2 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Carboxypeptidase b2 (cpb2) irna compositions and methods of use thereof
EP4013870A1 (en) 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof
PL4013385T3 (pl) 2019-08-14 2024-11-18 Acuitas Therapeutics, Inc. Ulepszone nanocząstki lipidowe do dostarczania kwasów nukleinowych
JP2022544412A (ja) 2019-08-14 2022-10-18 キュアバック アーゲー 免疫賦活特性が減少したrna組み合わせおよび組成物
PH12022550167A1 (en) 2019-08-16 2023-05-08 Regeneron Pharma High concentration anti-c5 formulations
EP4022058A2 (en) 2019-08-30 2022-07-06 Sixfold Bioscience Ltd. Compositions for transfer of cargo to cells
WO2021046122A1 (en) 2019-09-03 2021-03-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
CA3150452A1 (en) 2019-09-06 2021-03-11 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof
AU2020350759A1 (en) 2019-09-19 2022-03-31 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
EP4038189A1 (en) 2019-10-04 2022-08-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
US12486512B2 (en) 2019-10-18 2025-12-02 Alnylam Pharmaceuticals, Inc. Solute carrier family member iRNA compositions and methods of use thereof
JP7676377B2 (ja) 2019-10-22 2025-05-14 アルナイラム ファーマシューティカルズ, インコーポレイテッド 補体成分C3 iRNA組成物およびその使用方法
MX2022004712A (es) 2019-10-25 2022-08-04 Regeneron Pharma Regimenes de dosificacion para tratar o prevenir enfermedades asociadas a c5.
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
CN114728018B (zh) 2019-11-01 2024-07-19 阿尔尼拉姆医药品有限公司 亨廷顿(HTT)iRNA药剂组合物及其使用方法
KR20220115946A (ko) 2019-11-13 2022-08-19 알닐람 파마슈티칼스 인코포레이티드 안지오텐시노겐 (agt) 관련 장애를 치료하기 위한 방법 및 조성물
CN114728016B (zh) 2019-11-15 2024-10-29 富士胶片株式会社 脂质组合物
IL293051A (en) 2019-11-18 2022-07-01 Janssen Biotech Inc Vaccines based on mutant calr and jak2 and their uses
JP2023502576A (ja) 2019-11-22 2023-01-25 ジェネレーション バイオ カンパニー イオン化可能な脂質およびそれらのナノ粒子組成物
US20230056569A1 (en) 2019-11-22 2023-02-23 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
CA3164599A1 (en) 2019-12-13 2021-06-17 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
TW202138559A (zh) 2019-12-16 2021-10-16 美商阿尼拉製藥公司 含類PATATIN磷脂酶結構域3(PNPLA3)iRNA組成物及其使用方法
BR112022011803A2 (pt) 2019-12-20 2022-08-30 Curevac Ag Nanopartículas de lipídio para entrega de ácidos nucleicos
US12435035B2 (en) 2020-01-09 2025-10-07 Guide Therapeutics, Llc Nanomaterials
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
US12194089B2 (en) 2020-02-04 2025-01-14 CureVac SE Coronavirus vaccine
IL293571B2 (en) 2020-02-04 2025-01-01 Curevac Ag Coronavirus vaccine
KR20220140593A (ko) 2020-02-10 2022-10-18 알닐람 파마슈티칼스 인코포레이티드 Vegf-a 발현을 사일런싱하기 위한 조성물 및 방법
MX2022010052A (es) 2020-02-18 2022-09-05 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) contra la apolipoproteina c3 (apoc3) y metodos para su uso.
US12351834B2 (en) 2020-03-03 2025-07-08 Arcturus Therapeutics, Inc. Compositions and methods for the treatment of ornithine transcarbamylase deficiency
EP4114947A1 (en) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
JP2023516095A (ja) 2020-03-06 2023-04-17 アルナイラム ファーマシューティカルズ, インコーポレイテッド ケトヘキソキナーゼ(KHK)iRNA組成物およびその使用方法
WO2021183564A1 (en) 2020-03-09 2021-09-16 Arcturus Therapeutics, Inc. Compositions and methods for inducing immune responses
US20230134550A1 (en) 2020-03-24 2023-05-04 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
CN115667530A (zh) 2020-03-24 2023-01-31 世代生物公司 非病毒dna载体和其用于表达因子ix治疗剂的用途
TW202204615A (zh) 2020-03-26 2022-02-01 美商阿尼拉製藥公司 冠狀病毒iRNA組成物及其使用方法
KR20220160647A (ko) 2020-03-27 2022-12-06 제너레이션 바이오 컴퍼니 신규한 지질 및 이의 나노입자 조성물
WO2021202443A2 (en) 2020-03-30 2021-10-07 Alnylam Pharmaceucticals, Inc. Compositions and methods for silencing dnajc15 gene expression
KR20220161316A (ko) 2020-03-30 2022-12-06 비온테크 에스이 Claudin-18.2를 표적화하는 rna 조성물
US11773391B2 (en) 2020-04-01 2023-10-03 University of Pittsburgh—of the Commonwealth System of Higher Education Therapeutic and diagnostic target for SARS-CoV-2 and COVID-19
AR121769A1 (es) 2020-04-06 2022-07-06 Alnylam Pharmaceuticals Inc Composiciones y métodos para el silenciamiento de la expresión de myoc
BR112022020227A2 (pt) 2020-04-07 2022-11-22 Alnylam Pharmaceuticals Inc Composições e métodos para silenciamento da expressão de scn9a
WO2021206922A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
EP4133076A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Angiotensin-converting enzyme 2 (ace2) irna compositions and methods of use thereof
US12194157B2 (en) 2020-04-09 2025-01-14 Finncure Oy Carrier for targeted delivery to a host
WO2021205077A1 (en) 2020-04-09 2021-10-14 Finncure Oy Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses
CN118490818A (zh) * 2020-04-22 2024-08-16 生物技术欧洲股份公司 冠状病毒疫苗
KR20230018377A (ko) 2020-04-27 2023-02-07 알닐람 파마슈티칼스 인코포레이티드 아포지질단백질 e (apoe) irna 제제 조성물 및 이의 사용 방법
WO2021220053A2 (en) 2020-04-27 2021-11-04 Sixfold Bioscience Ltd. Compositions containing nucleic acid nanoparticles with modular functionality
MX2022013606A (es) 2020-04-30 2023-01-16 Alnylam Pharmaceuticals Inc Composiciones de ácido ribonucleico interferente (arni) del factor b de complemento (cfb) y métodos de uso de las mismas.
JP2023524071A (ja) 2020-05-01 2023-06-08 アークトゥラス・セラピューティクス・インコーポレイテッド 嚢胞性線維症を治療するための核酸及び方法
CA3162416C (en) 2020-05-15 2023-07-04 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
EP4150086A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
EP4150078A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
EP4150087A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
EP4150090A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021231680A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
WO2021231685A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
CA3179444A1 (en) 2020-05-20 2021-11-25 Avak Kahvejian Immunogenic compositions and uses thereof
AU2021275223A1 (en) 2020-05-20 2023-02-02 Flagship Pioneering Innovations Vi, Llc. Coronavirus antigen compositions and their uses
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
KR20230029685A (ko) 2020-05-29 2023-03-03 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 Trem 조성물 및 이에 관련된 방법
WO2021243301A2 (en) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc. Trem compositions and methods relating thereto
CA3170740A1 (en) 2020-05-29 2021-12-02 Curevac Ag Nucleic acid based combination vaccines
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
WO2021252557A1 (en) 2020-06-09 2021-12-16 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
EP4162033A4 (en) 2020-06-09 2024-12-25 Alnylam Pharmaceuticals, Inc. Sirna compositions and methods for silencing gpam (glycerol-3-phosphate acyltransferase 1, mitochondrial) expression
IL299094A (en) 2020-06-15 2023-02-01 Res Inst Nationwide Childrens Hospital Administration of an adeno-associated virus vector for muscular dystrophies
BR112022024420A2 (pt) 2020-06-18 2023-01-17 Alnylam Pharmaceuticals Inc Composições de irna de xantina desidrogenase (xdh) e métodos de uso das mesmas
WO2025056938A1 (en) 2023-09-11 2025-03-20 BioNTech SE Rna compositions for delivery of incretin agents
US12295997B2 (en) 2020-07-06 2025-05-13 Janssen Biotech, Inc. Prostate neoantigens and their uses
CA3188801A1 (en) 2020-07-08 2022-01-13 Janssen Sciences Ireland Unlimited Company Rna replicon vaccines against hbv
US20240065982A1 (en) * 2020-07-10 2024-02-29 Genevant Sciences Gmbh Lipid nanoparticles for delivering therapeutics to lungs
BR112023000327A2 (pt) 2020-07-16 2023-01-31 Acuitas Therapeutics Inc Lipídeos catiônicos para o uso em nanopartículas lipídicas
BR112023001648A2 (pt) 2020-07-27 2023-04-04 Anjarium Biosciences Ag Moléculas de dna de fita dupla, veículo de entrega e método para preparar uma molécula de dna com extremidade em grampo
CA3170741A1 (en) 2020-07-31 2022-02-03 Curevac Ag Nucleic acid encoded antibody mixtures
JP2023537609A (ja) 2020-08-14 2023-09-04 アークトゥラス・セラピューティクス・インコーポレイテッド 脂質ナノ粒子を凍結乾燥する方法
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
EP4157344A2 (en) 2020-08-31 2023-04-05 CureVac SE Multivalent nucleic acid based coronavirus vaccines
AU2021336976A1 (en) 2020-09-03 2023-03-23 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
EP4212162A4 (en) 2020-09-14 2024-02-28 FUJIFILM Corporation LIPID COMPOSITION
WO2022060871A1 (en) 2020-09-15 2022-03-24 Verve Therapeutics, Inc. Lipid formulations for gene editing
TW202228784A (zh) 2020-09-23 2022-08-01 奧地利商艾柏力維亞生技有限公司 用以於一患者中螯合非預期的抗peg抗體的化合物
WO2022066847A1 (en) 2020-09-24 2022-03-31 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
WO2022076291A1 (en) 2020-10-05 2022-04-14 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
BR112023006710A2 (pt) 2020-10-14 2023-10-03 George Mason Res Foundation Inc Métodos de fabricação de nanopartícula lipídica e composições derivadas da mesma
AU2021365822A1 (en) 2020-10-21 2023-06-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
WO2022087329A1 (en) 2020-10-23 2022-04-28 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
TW202233232A (zh) 2020-11-06 2022-09-01 法商賽諾菲公司 遞送mRNA疫苗的脂質奈米顆粒
KR20230107625A (ko) 2020-11-13 2023-07-17 알닐람 파마슈티칼스 인코포레이티드 응고 인자 V(F5) iRNA 조성물 및 이의 사용 방법
IL303195A (en) 2020-11-25 2023-07-01 Akagera Medicines Inc Lipid nanoparticles for delivery of nucleic acids and related methods of use
WO2022119873A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
PE20231178A1 (es) 2020-12-03 2023-08-01 Scribe Therapeutics Inc Sistemas crispr tipo v clase 2 disenados por ingenieria
EP4259795A1 (en) 2020-12-08 2023-10-18 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
WO2022140252A1 (en) 2020-12-21 2022-06-30 Beam Therapeutics Inc. Nanomaterials comprising ester-linked acetals
KR20230164648A (ko) 2020-12-22 2023-12-04 큐어백 에스이 SARS-CoV-2 변이체에 대한 RNA 백신
IL303855A (en) * 2020-12-22 2023-08-01 Univ Cornell Zwitterionic lipid nanoparticle compositions, and methods of use
CA3171051A1 (en) 2020-12-22 2022-06-30 Curevac Ag Pharmaceutical composition comprising lipid-based carriers encapsulating rna for multidose administration
WO2022137133A1 (en) 2020-12-22 2022-06-30 Curevac Ag Rna vaccine against sars-cov-2 variants
KR20230135585A (ko) 2020-12-23 2023-09-25 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 변형된 trem의 조성물 및 이의 용도
TWI869643B (zh) * 2020-12-28 2025-01-11 財團法人工業技術研究院 免疫刺激性脂質複合體、包含免疫刺激性脂質複合體的醫藥組合物以及其用途
JP2024501022A (ja) 2020-12-28 2024-01-10 アークトゥルス セラピューティクス, インコーポレイテッド Hbvを標的とする転写活性化因子様エフェクターヌクレアーゼ(talen)
EP4274896A1 (en) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Complement component 9 (c9) irna compositions and methods of use thereof
AU2022210313A1 (en) 2021-01-20 2023-06-29 Beam Therapeutics Inc. Nanomaterials
EP4281080A4 (en) 2021-01-20 2025-09-24 Beam Therapeutics Inc NANOMATERIALS COMPRISING A BIODEGRADABLE ELEMENT
MX2023007788A (es) 2021-01-24 2023-11-17 Michael David Forrest Inhibidores de la atp sintasa, usos cosmeticos y terapeuticos.
EP4087938A2 (en) 2021-01-27 2022-11-16 CureVac AG Method of reducing the immunostimulatory properties of in vitro transcribed rna
TW202245835A (zh) 2021-02-04 2022-12-01 美商默沙東有限責任公司 用於肺炎球菌結合物疫苗之奈米乳化液佐劑組合物
WO2022174000A2 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
EP4298220A1 (en) 2021-02-25 2024-01-03 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods of use thereof
JP2024507394A (ja) 2021-02-26 2024-02-19 エスリス ゲーエムベーハー エアロゾル形成用の製剤および核酸を送達するためのエアロゾル
JP2024515423A (ja) 2021-02-26 2024-04-10 アルナイラム ファーマシューティカルズ, インコーポレイテッド ケトヘキソキナーゼ(KHK)iRNA組成物およびその使用方法
CA3212128A1 (en) 2021-03-04 2022-09-09 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
CA3214137A1 (en) 2021-03-26 2022-09-29 Mina Therapeutics Limited Tmem173 sarna compositions and methods of use
JP2024511206A (ja) 2021-03-26 2024-03-12 グラクソスミスクライン バイオロジカルズ ソシエテ アノニム 免疫原性組成物
CN117203336A (zh) 2021-03-29 2023-12-08 阿尔尼拉姆医药品有限公司 亨廷顿蛋白(HTT)iRNA药剂组合物以及其使用方法
US20250345524A1 (en) 2021-03-31 2025-11-13 CureVac SE Syringes containing pharmaceutical compositions comprising rna
JP2024512669A (ja) 2021-03-31 2024-03-19 フラグシップ パイオニアリング イノベーションズ ブイ,インコーポレーテッド タノトランスミッションポリペプチド及び癌の処置におけるそれらの使用
WO2022212153A1 (en) 2021-04-01 2022-10-06 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
DK4319803T3 (da) 2021-04-08 2025-12-15 Vaxthera Sas Coronavirusvaccine omfattende et mosaikprotein
KR20240012370A (ko) 2021-04-20 2024-01-29 안자리움 바이오사이언시스 아게 아밀로-알파-1, 6-글루코시다제, 4-알파-글루카노트랜스퍼라제를 인코딩하는 dna 분자의 조성물, 이를 제조하는 방법 및 이를 사용하는 방법
WO2022225368A1 (ko) 2021-04-22 2022-10-27 (주)인벤티지랩 지질 나노입자의 제조 방법 및 이의 제조 장치
CA3216106A1 (en) 2021-04-26 2022-11-03 Alnylam Pharmaceuticals, Inc. Transmembrane protease, serine 6 (tmprss6) irna compositions and methods of use thereof
WO2022232286A1 (en) 2021-04-27 2022-11-03 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
MX2023012643A (es) 2021-04-27 2024-01-05 Generation Bio Co Vectores de adn no virales que expresan anticuerpos terapéuticos y usos de estos.
WO2022232343A1 (en) 2021-04-29 2022-11-03 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
EP4334446A1 (en) 2021-05-03 2024-03-13 CureVac SE Improved nucleic acid sequence for cell type specific expression
WO2022245583A1 (en) 2021-05-18 2022-11-24 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
WO2022246023A1 (en) 2021-05-20 2022-11-24 Korro Bio, Inc. Methods and compositions for adar-mediated editing
WO2022244831A1 (en) 2021-05-21 2022-11-24 Okinawa Institute Of Science And Technology School Corporation Coronavirus vaccine suitable for elderly individuals
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
EP4347823A1 (en) 2021-06-02 2024-04-10 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
BR112023025224A2 (pt) 2021-06-04 2024-02-27 Alnylam Pharmaceuticals Inc Quadro de leitura aberto 72 do cromossomo humano 9 (c9orf72) composições de agente de irna e métodos de uso dos mesmos
WO2022260939A2 (en) 2021-06-08 2022-12-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
CA3173953A1 (en) 2021-06-11 2023-12-10 Tyson D. BOWEN Rna polymerase iii promoters and methods of use
CN117460542A (zh) 2021-06-11 2024-01-26 纳米智能生物医学工程有限公司 包含用于将寡核苷酸递送至靶细胞内的肽-脂质缀合物的纳米颗粒以及包含其的药物组合物
EP4355727A1 (en) 2021-06-14 2024-04-24 Generation Bio Co. Cationic lipids and compositions thereof
MX2023014901A (es) 2021-06-22 2024-04-29 Bio Trip B V Nanoparticulas que contienen acido nucleico.
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
US20250034564A1 (en) 2021-06-29 2025-01-30 Korro Bio, Inc. Methods and Compositions for ADAR-Mediated Editing
IL309296A (en) 2021-06-30 2024-02-01 Alnylam Pharmaceuticals Inc Methods and compositions for the treatment of angiotensinogen-related disorder (AGT-)
US20250270562A1 (en) 2021-07-09 2025-08-28 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
US20230044997A1 (en) 2021-07-15 2023-02-09 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs and methods of use for cell rejuvenation and for treatment
US20230042860A1 (en) 2021-07-15 2023-02-09 Turn Biotechnologies, Inc. Polycistronic expression vectors
WO2023288288A1 (en) 2021-07-15 2023-01-19 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs with on/off mechanism for controlled expression and methods of use
TW202421169A (zh) 2021-07-21 2024-06-01 美商艾拉倫製藥股份有限公司 代謝疾患相關的標靶基因iRNA組成物及其使用方法
AU2022316139A1 (en) 2021-07-23 2024-01-18 Alnylam Pharmaceuticals, Inc. Beta-catenin (ctnnb1) irna compositions and methods of use thereof
EP4377457A1 (en) 2021-07-26 2024-06-05 Flagship Pioneering Innovations VI, LLC Trem compositions and uses thereof
US20250258180A1 (en) 2021-07-27 2025-08-14 Stand Therapeutics Co., Ltd. Peptide tag and nucleic acid encoding same
EP4377458A1 (en) 2021-07-29 2024-06-05 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
WO2023006999A2 (en) 2021-07-30 2023-02-02 CureVac SE Mrnas for treatment or prophylaxis of liver diseases
KR20240042004A (ko) 2021-08-03 2024-04-01 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴(TTR) iRNA 조성물 및 이의 사용 방법
PE20241132A1 (es) 2021-08-04 2024-05-24 Alnylam Pharmaceuticals Inc Composiciones de arni y metodos para silenciar el angiotensinogeno (agt)
WO2023019246A1 (en) 2021-08-13 2023-02-16 Alnylam Pharmaceuticals, Inc. Factor xii (f12) irna compositions and methods of use thereof
WO2023023055A1 (en) 2021-08-16 2023-02-23 Renagade Therapeutics Management Inc. Compositions and methods for optimizing tropism of delivery systems for rna
MX2024002440A (es) 2021-08-31 2024-03-08 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) del efector b similar al factor de fragmentacion de adn subunidad alfa (dffa) que induce la muerte celular (cideb) y metodos de uso de estas.
WO2023031394A1 (en) 2021-09-03 2023-03-09 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
MX2024002725A (es) 2021-09-03 2024-03-15 CureVac SE Nuevas nanoparticulas lipidicas para la administracion de acidos nucleicos que comprenden fosfatidilserina.
WO2023044333A1 (en) 2021-09-14 2023-03-23 Renagade Therapeutics Management Inc. Cyclic lipids and methods of use thereof
JP2024534697A (ja) 2021-09-14 2024-09-20 レナゲード セラピューティクス マネージメント インコーポレイテッド 非環状脂質及びその使用方法
EP4401742A2 (en) 2021-09-17 2024-07-24 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
EP4464783A3 (en) 2021-09-17 2025-01-22 Flagship Pioneering Innovations VI, LLC Compositions and methods for producing circular polyribonucleotides
IL311454A (en) 2021-09-20 2024-05-01 Alnylam Pharmaceuticals Inc Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
CA3231909A1 (en) 2021-09-21 2023-03-30 Jason Fernandes Engineered casx repressor systems
EP4416284A1 (en) 2021-10-15 2024-08-21 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
TW202322826A (zh) 2021-10-18 2023-06-16 美商旗艦先鋒創新有限責任公司 用於純化多核糖核苷酸之組成物及方法
WO2023069603A1 (en) 2021-10-22 2023-04-27 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
EP4423273A1 (en) 2021-10-29 2024-09-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
TW202334418A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 杭丁頓(HTT)iRNA劑組成物及其使用方法
EP4422698A1 (en) 2021-10-29 2024-09-04 CureVac SE Improved circular rna for expressing therapeutic proteins
US20250043269A1 (en) 2021-11-03 2025-02-06 The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glad Precise Genome Editing Using Retrons
KR20240107139A (ko) 2021-11-08 2024-07-08 오나 테라퓨틱스, 인코포레이티드 원형 폴리뉴클레오티드를 전달하기 위한 지질 나노입자 조성물
KR102772764B1 (ko) 2021-11-10 2025-02-27 주식회사 에스엠엘바이오팜 트레할로즈 유도체 및 신규 구조 유지 화합물을 포함하는 핵산 의약품 전달용 지질나노입자의 약학 조성물
US20250049948A1 (en) 2021-11-16 2025-02-13 Sail Biomedicines, Inc. Novel ionizable lipids and lipid nanoparticles and methods of using the same
KR20240128683A (ko) 2021-11-22 2024-08-26 세일 바이오메디슨스, 인크. 신규한 이온화 가능 지질 및 지질 나노입자, 및 이를 사용하는 방법
EP4436598A2 (en) 2021-11-24 2024-10-02 Flagship Pioneering Innovations VI, LLC Immunogenic compositions and their uses
IL313004A (en) 2021-11-24 2024-07-01 Flagship Pioneering Innovations Vi Llc Coronavirus immunogen compositions and their uses
IL312799A (en) 2021-11-24 2024-07-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions of varicella-zoster virus and uses thereof
US12186387B2 (en) 2021-11-29 2025-01-07 BioNTech SE Coronavirus vaccine
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
KR20240123832A (ko) 2021-12-16 2024-08-14 아퀴타스 테라퓨틱스 인크. 지질 나노입자 제형에 사용하기 위한 지질
EP4448758A1 (en) 2021-12-17 2024-10-23 Flagship Pioneering Innovations VI, LLC Methods for enrichment of circular rna under denaturing conditions
EP4452925A1 (en) 2021-12-20 2024-10-30 Beam Therapeutics Inc. Nanomaterial comprising diamines
EP4452942A1 (en) 2021-12-20 2024-10-30 Beam Therapeutics Inc. Nanomaterials comprising disulfides
EP4452930A1 (en) 2021-12-20 2024-10-30 Beam Therapeutics Inc. Ionizable amine and ester lipids and lipid nanoparticles
CA3241503A1 (en) 2021-12-20 2023-06-29 Beam Therapeutics Inc. Nanomaterials comprising tetravalent lipid compounds
JP2025501731A (ja) 2021-12-20 2025-01-23 ビーム セラピューティクス インク. イオン化可能なアミン脂質及び脂質ナノ粒子
CA3241061A1 (en) 2021-12-22 2023-06-29 Alexandra Sophie DE BOER Compositions and methods for purifying polyribonucleotides
US20230201817A1 (en) 2021-12-23 2023-06-29 Biontech Delivery Technologies Gmbh Modular drug production system
CA3243054A1 (en) 2021-12-23 2025-04-08 Renagade Therapeutics Management Inc. Constrained Lipids and Associated Utilization Processes
MX2024007870A (es) 2021-12-23 2024-08-15 Flagship Pioneering Innovations Vi Llc Polirribonucleotidos circulares que codifican polipeptidos antifusogenicos.
US20250381294A1 (en) 2022-01-14 2025-12-18 Anjarium Biosciences Ag Compositions of DNA Molecules Encoding Factor VIII, Methods of Making Thereof, and Methods of Use Thereof
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023144193A1 (en) 2022-01-25 2023-08-03 CureVac SE Mrnas for treatment of hereditary tyrosinemia type i
WO2023145755A1 (ja) 2022-01-25 2023-08-03 公益財団法人川崎市産業振興財団 経皮投与用のrnaを含む組成物および当該組成物の投与方法
US20250345416A1 (en) 2022-01-27 2025-11-13 BioNTech SE Pharmaceutical compositions for delivery of herpes simplex virus antigens and related methods
WO2023144330A1 (en) 2022-01-28 2023-08-03 CureVac SE Nucleic acid encoded transcription factor inhibitors
WO2023161350A1 (en) 2022-02-24 2023-08-31 Io Biotech Aps Nucleotide delivery of cancer therapy
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
KR20240161965A (ko) 2022-03-14 2024-11-13 제너레이션 바이오 컴퍼니 이종 프라임 부스트 백신 조성물 및 사용 방법
EP4494644A4 (en) 2022-03-15 2025-06-11 FUJIFILM Corporation LIPID COMPOSITION
KR20240157096A (ko) 2022-03-23 2024-10-31 나노베이션 테라퓨틱스 인크. 고 스테롤-함유 지질 나노입자
KR20240166554A (ko) 2022-03-25 2024-11-26 세일 바이오메디슨스, 인크. 신규한 이온화 가능 지질 및 지질 나노입자, 및 이를 사용하는 방법
US12011507B2 (en) 2022-04-01 2024-06-18 Nanovation Therapeutics Inc. MRNA delivery composition
CN119562762A (zh) 2022-04-04 2025-03-04 加利福尼亚大学董事会 遗传互补组合物和方法
CA3255619A1 (en) 2022-04-07 2023-10-12 Renagade Therapeutics Management Inc. CYCLIC LIPIDS AND LIPID NANOPARTICLES (LPNs) FOR THE SUPPLY OF NUCLEIC ACIDS OR PEPTIDES INTENDED FOR USE IN VACCINATION AGAINST INFECTIOUS AGENTS
WO2023196634A2 (en) 2022-04-08 2023-10-12 Flagship Pioneering Innovations Vii, Llc Vaccines and related methods
WO2023199943A1 (ja) 2022-04-12 2023-10-19 国立研究開発法人理化学研究所 コロナウイルス感染症を処置することに用いられる抗体
WO2023198828A1 (en) 2022-04-13 2023-10-19 Universitat Autònoma De Barcelona Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
AU2023260227A1 (en) 2022-04-27 2024-11-21 Shanghai Regenelead Therapies Co., Ltd Nucleic acid construct and use thereof
EP4522743A1 (en) 2022-05-09 2025-03-19 Flagship Pioneering Innovations VI, LLC Trem compositions and methods of use for treating proliferative disorders
IL316630A (en) 2022-05-13 2024-12-01 BioNTech SE HIV-targeted RNA complexes
JP2025516638A (ja) 2022-05-13 2025-05-30 フラッグシップ パイオニアリング イノベーションズ セブン,エルエルシー 二本鎖dna組成物及び関連する方法
WO2023218420A1 (en) 2022-05-13 2023-11-16 Janssen Pharmaceuticals, Inc. Mrna compositions for inducing latent hiv-1 reversal
WO2023217267A1 (zh) 2022-05-13 2023-11-16 上海瑞宏迪医药有限公司 包含utr的核酸构建体及其应用
WO2023230098A1 (en) 2022-05-23 2023-11-30 Logicbio Therapeutics, Inc. Gene therapy compositions and methods of use thereof
JP2025522297A (ja) 2022-05-25 2025-07-15 ビオンテック エスエー サルポックス抗原の送達のためのrna組成物及び関連する方法
CN119212720A (zh) 2022-05-25 2024-12-27 库瑞瓦格欧洲股份公司 编码大肠杆菌FimH抗原性多肽的基于核酸的疫苗
JP2025522311A (ja) 2022-05-25 2025-07-15 アカゲラ・メディスンズ,インコーポレイテッド 核酸を送達するための脂質ナノ粒子およびその使用方法
JP2025518221A (ja) 2022-05-30 2025-06-12 ビオンテック・ソシエタス・エウロパエア 核酸の送達のための複合体
AU2023278164A1 (en) 2022-06-02 2024-12-05 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
IL317412A (en) 2022-06-03 2025-02-01 Bio Trip B V Lipid nanoparticles based on multifunctional molecules for nucleic acid delivery
EP4536819A1 (en) 2022-06-07 2025-04-16 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2023239756A1 (en) 2022-06-07 2023-12-14 Generation Bio Co. Lipid nanoparticle compositions and uses thereof
GB2634837A (en) 2022-06-07 2025-04-23 Scribe Therapeutics Inc Compositions and methods for the targeting of PCSK9
WO2023250112A1 (en) 2022-06-22 2023-12-28 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
WO2024002985A1 (en) 2022-06-26 2024-01-04 BioNTech SE Coronavirus vaccine
EP4558624A2 (en) 2022-07-18 2025-05-28 Renagade Therapeutics Management Inc. Gene editing components, systems, and methods of use
JP2025528020A (ja) 2022-07-20 2025-08-26 ビーム セラピューティクス インク. トリオールを含むナノ材料
AU2023320333A1 (en) 2022-08-01 2025-01-16 Flagship Pioneering Innovations Vii, Llc Immunomodulatory proteins and related methods
EP4569112A1 (en) 2022-08-12 2025-06-18 Remix Therapeutics Inc. Methods and compositions for modulating splicing at alternative splice sites
CN120112633A (zh) 2022-08-12 2025-06-06 生命编辑治疗股份有限公司 Rna引导的核酸酶及其活性片段和变体以及使用方法
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
EP4577243A1 (en) 2022-08-26 2025-07-02 Ethris GmbH Stable lipid or lipidoid nanoparticle suspensions
EP4327829A1 (en) 2022-08-26 2024-02-28 Ethris GmbH Stabilization of lipid or lipidoid nanoparticle suspensions
KR20250056256A (ko) 2022-08-31 2025-04-25 세일 바이오메디슨스, 인크. 신규 이온화성 지질 및 지질 나노입자, 그리고 이의 사용 방법
CN120152962A (zh) 2022-09-06 2025-06-13 国立大学法人九州大学 脂质纳米粒子
WO2024059165A1 (en) 2022-09-15 2024-03-21 Alnylam Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
EP4342460A1 (en) 2022-09-21 2024-03-27 NovoArc GmbH Lipid nanoparticle with nucleic acid cargo
WO2024063788A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
EP4590330A1 (en) 2022-09-23 2025-07-30 BioNTech SE Compositions for delivery of liver stage antigens and related methods
AU2023347390A1 (en) 2022-09-23 2025-04-10 BioNTech SE Compositions for delivery of plasmodium csp antigens and related methods
EP4590822A1 (en) 2022-09-23 2025-07-30 nChroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024063789A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
KR20250075664A (ko) 2022-09-26 2025-05-28 글락소스미스클라인 바이오로지칼즈 에스.에이. 인플루엔자 바이러스 백신
WO2024068728A1 (en) 2022-09-27 2024-04-04 Resolution Therapeutics Limited Enhanced macrophages
WO2024073588A1 (en) * 2022-09-28 2024-04-04 Board Of Regents, The University Of Texas System Respiratory dry powder delivery
WO2024071319A1 (en) 2022-09-28 2024-04-04 Okinawa Institute Of Science And Technology School Corporation Composition comprising isolated or enriched phosphoenolpyruvate or a salt thereof
WO2024077191A1 (en) 2022-10-05 2024-04-11 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additionalpolypeptides and their use in treating cancer
JP2025534447A (ja) 2022-10-06 2025-10-15 バイオエヌテック エスエー クローディン18.2を標的とするrna組成物
WO2024074211A1 (en) 2022-10-06 2024-04-11 BioNTech SE Rna compositions targeting claudin-18.2
WO2024085149A1 (ja) 2022-10-18 2024-04-25 第一三共株式会社 テイラー反応装置及びカプセル粒子の製造方法
WO2024083345A1 (en) 2022-10-21 2024-04-25 BioNTech SE Methods and uses associated with liquid compositions
DE202023106198U1 (de) 2022-10-28 2024-03-21 CureVac SE Impfstoff auf Nukleinsäurebasis
EP4612296A1 (en) 2022-10-31 2025-09-10 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
WO2024102730A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and nanoparticle compositions for delivering polynucleotides
TW202428289A (zh) 2022-11-08 2024-07-16 美商歐納醫療公司 環狀rna組合物
AR131008A1 (es) 2022-11-08 2025-02-05 Flagship Pioneering Innovations Vi Llc Composiciones y métodos para producir polirribonucleótidos circulares
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024102954A1 (en) 2022-11-10 2024-05-16 Massachusetts Institute Of Technology Activation induced clipping system (aics)
CN120641465A (zh) 2022-12-01 2025-09-12 世代生物公司 新颖的聚甘油缀合脂质和包含其的脂质纳米颗粒组合物
EP4626402A1 (en) 2022-12-01 2025-10-08 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
KR20250114116A (ko) 2022-12-01 2025-07-28 제너레이션 바이오 컴퍼니 세포 표적화를 위한 스텔스 지질 나노입자 조성물
KR20250129819A (ko) 2022-12-01 2025-08-29 제너레이션 바이오 컴퍼니 핵산, 이온화 가능한 지질, 스테롤, 지질에 고정된 중합체, 및 헬퍼 지질을 포함하는 지질 나노입자 및 이의 용도
KR20240087591A (ko) 2022-12-09 2024-06-19 주식회사 나이벡 타겟 세포 내로 올리고뉴클레오티드를 전달하기 위한 펩타이드 기반 결합체를 포함하는 나노입자 및 이를 포함하는 약학적 조성물
WO2024123134A1 (ko) 2022-12-09 2024-06-13 서울대학교산학협력단 B 세포 및 t 세포 내로 mrna를 전달하기 위한 펩타이드 기반 결합체를 포함하는 나노입자 및 이의 용도
TW202430215A (zh) 2022-12-14 2024-08-01 美商旗艦先鋒創新有限責任(Vii)公司 用於將治療劑遞送至骨之組成物和方法
TW202440929A (zh) 2022-12-14 2024-10-16 加拿大商普羅維登斯治療控股公司 用於感染性疾病的組合物和方法
WO2024129982A2 (en) 2022-12-15 2024-06-20 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024133486A1 (en) 2022-12-20 2024-06-27 Universiteit Utrecht Holding B.V. Nucleic acid-lipid particles
WO2024134199A1 (en) 2022-12-22 2024-06-27 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
CN121127490A (zh) 2022-12-28 2025-12-12 生物技术欧洲股份公司 靶向hiv的rna组合物
WO2024141786A2 (en) 2022-12-29 2024-07-04 Popvax Private Limited Multitarget vaccines and therapeutics
WO2024141784A2 (en) 2022-12-29 2024-07-04 Popvax Private Limited Broadly protective betacoronavirus vaccines and compositions
CN120529963A (zh) 2023-01-04 2025-08-22 创技公司株式会社 具有螺旋结构的用于制备脂质纳米粒子的芯片及利用其制备脂质纳米粒子的方法
WO2024151685A1 (en) 2023-01-09 2024-07-18 Beth Israel Deaconess Medical Center, Inc. Recombinant nucleic acid molecules and their use in wound healing
US20240238473A1 (en) 2023-01-09 2024-07-18 Beth Israel Deaconess Medical Center, Inc. Recombinant nucleic acid molecules and their use in wound healing
WO2024151687A1 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations V, Inc. Genetic switches and their use in treating cancer
EP4648794A2 (en) 2023-01-09 2025-11-19 Flagship Pioneering Innovations VII, LLC Vaccines and related methods
EP4652262A1 (en) 2023-01-19 2025-11-26 Altos Labs, Inc. Use of regeneration factors in organ transplantation
EP4658239A1 (en) 2023-02-03 2025-12-10 GlaxoSmithKline Biologicals S.A. Rna formulation
TW202438515A (zh) 2023-02-06 2024-10-01 美商旗艦先鋒創新有限責任(Vii)公司 免疫調節組合物及相關方法
IL322261A (en) 2023-02-09 2025-09-01 Alnylam Pharmaceuticals Inc Reverser molecules and methods of using them
EP4665736A2 (en) 2023-02-13 2025-12-24 Flagship Pioneering Innovations VII, LLC Cleavable linker-containing ionizable lipids and lipid carriers for therapeutic compositions
GB202302092D0 (en) 2023-02-14 2023-03-29 Glaxosmithkline Biologicals Sa Analytical method
IL322201A (en) 2023-02-17 2025-09-01 Flagship Pioneering Innovations Vii Llc DNA structures containing modified uracil
EP4665856A2 (en) 2023-02-17 2025-12-24 Flagship Pioneering Innovations VII, LLC Dna compositions comprising modified cytosine
AU2024233180A1 (en) 2023-03-08 2025-09-25 CureVac SE Novel lipid nanoparticle formulations for delivery of nucleic acids
AU2024234874A1 (en) 2023-03-15 2025-10-09 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
AU2024235803A1 (en) 2023-03-15 2025-09-25 Flagship Pioneering Innovations Vi, Llc Compositions comprising polyribonucleotides and uses thereof
AU2024236558A1 (en) 2023-03-15 2025-10-09 Renagade Therapeutics Management Inc. Delivery of gene editing systems and methods of use thereof
WO2024192422A1 (en) 2023-03-15 2024-09-19 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
EP4570269A4 (en) 2023-03-15 2025-10-22 United Immunity Co Ltd LIPID PARTICLE
WO2024194767A1 (en) 2023-03-17 2024-09-26 BioNTech SE Automated drug production system
AU2024248139A1 (en) 2023-03-29 2025-09-25 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2024206620A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Messenger rna encoding casx
TW202444914A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 抑制子融合蛋白系統
WO2024206676A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of lpa
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024209418A1 (en) 2023-04-07 2024-10-10 BioNTech SE Heating and cooling jacket for fluid vessels
AU2024255972A1 (en) 2023-04-12 2025-10-23 Flagship Pioneering Innovations Vi, Llc Modified trems, compositions, and related methods thereof
WO2024216128A1 (en) 2023-04-12 2024-10-17 Flagship Pioneering Innovations Vi, Llc Trems for use in correction of missense mutations
WO2024213740A1 (en) 2023-04-14 2024-10-17 BioNTech SE Vaccine mixing method, syringe and system
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
CN121001738A (zh) 2023-04-27 2025-11-21 葛兰素史克生物有限公司 流感病毒疫苗
WO2024223724A1 (en) 2023-04-27 2024-10-31 Glaxosmithkline Biologicals Sa Influenza virus vaccines
CN121038807A (zh) 2023-05-03 2025-11-28 生物技术欧洲股份公司 优化的csp变体及相关方法
US20240401025A1 (en) 2023-05-03 2024-12-05 Manifold Biotechnologies, Inc. Methods and compositions for high-throughput protein delivery, screening, and detection
WO2024228044A1 (en) 2023-05-03 2024-11-07 BioNTech SE Optimized csp variants and related methods
AU2024269222A1 (en) 2023-05-05 2025-10-09 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
DE102023001946A1 (de) 2023-05-12 2024-11-14 Friedrich-Schiller-Universität Jena, Körperschaft des öffentlichen Rechts Nanopartikel für den Transport von Wirkstoffen mit anionischen Gruppen, Verfahren zu deren Herstellung und deren Verwendung
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024238700A1 (en) 2023-05-15 2024-11-21 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024240962A1 (en) 2023-05-25 2024-11-28 Ethris Gmbh Gm-csf-encoding nucleic acids, pharmaceutical compositions, methods and uses thereof
WO2024249954A1 (en) 2023-05-31 2024-12-05 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2024258829A1 (en) 2023-06-12 2024-12-19 Flagship Pioneering Innovations Vii, Llc Sars-cov-2 vaccine compositions and related methods
WO2025006684A1 (en) 2023-06-28 2025-01-02 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
WO2025003406A1 (en) 2023-06-29 2025-01-02 BioNTech SE Modular multiplatform system for mrna drug production
WO2025007148A1 (en) 2023-06-30 2025-01-02 Orna Therapeutics, Inc. Polymer lipid nanoparticle compositions for delivering circular polynucleotides
TW202517669A (zh) 2023-07-05 2025-05-01 日商武田藥品工業股份有限公司 用於A型血友病之基因療法的編碼表現增加之重組FVlll變異體的病毒載體
TW202515531A (zh) 2023-07-07 2025-04-16 美商輝瑞股份有限公司 兩親性tlr7/8佐劑及其用途
WO2025011529A2 (en) 2023-07-07 2025-01-16 Shanghai Circode Biomed Co., Ltd. Circular rna vaccines for seasonal flu and methods of uses
WO2025015335A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025015338A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025024324A1 (en) 2023-07-21 2025-01-30 BioNTech SE Compositions for delivery of plasmodium antigens and related methods
WO2025024335A2 (en) 2023-07-21 2025-01-30 BioNTech SE Compositions for delivery of plasmodium antigens and related methods
WO2025024337A1 (en) 2023-07-24 2025-01-30 BioNTech SE Compositions for delivery of plasmodium antigens and related methods
WO2025021083A1 (en) * 2023-07-24 2025-01-30 National Institute Of Biological Sciences, Beijing Lipid nanoparticle compositions for localized treatment of skin diseases
TW202516001A (zh) 2023-07-25 2025-04-16 美商旗艦先鋒創新有限責任(Vii)公司 Cas內切酶及相關方法
WO2025022367A2 (en) 2023-07-27 2025-01-30 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
WO2025026545A1 (en) 2023-08-01 2025-02-06 BioNTech SE Ionizable thioplipids and uses thereof
TW202519509A (zh) 2023-08-01 2025-05-16 德商拜恩技術股份公司 可離子化硫脂質及其用途
WO2025027576A2 (en) 2023-08-03 2025-02-06 BioNTech SE Rna compositions targeting hiv
NL2035538B1 (en) 2023-08-03 2025-02-18 Univ Eindhoven Tech Lipid-like copolymers and method of producing these and the use thereof, pharmaceutical compositions comprising said copolymers, and the use of said compositions.
WO2025030154A1 (en) 2023-08-03 2025-02-06 The Trustees Of The University Of Pennsylvania Pharmaceutical compositions for delivery of herpes simplex virus glycoprotein b antigens and related methods
WO2025030097A2 (en) 2023-08-03 2025-02-06 BioNTech SE Pharmaceutical compositions for delivery of herpes simplex virus antigens and related methods
WO2025027579A2 (en) 2023-08-03 2025-02-06 BioNTech SE Rna compositions targeting hiv
WO2025034422A1 (en) 2023-08-04 2025-02-13 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating ctnnb1-associated disorders
US12162927B1 (en) 2023-08-09 2024-12-10 Wyvern Pharmaceuticals Inc. Compositions and methods for regulating production of an antibody like protein and ribonucleic acid
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025045767A1 (en) 2023-08-25 2025-03-06 Ethris Gmbh Stabilized lipid and lipidoid nanoparticle formulations with specific surfactant properties for enhanced pharmaceutical applications
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
WO2025045142A1 (en) 2023-08-29 2025-03-06 Shanghai Circode Biomed Co., Ltd. Circular rna encoding vegf polypeptides, formulations, and methods of uses
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025046121A1 (en) 2023-09-01 2025-03-06 Novoarc Gmbh Lipid nanoparticle with nucleic acid cargo and ionizable lipid
EP4520345A1 (en) 2023-09-06 2025-03-12 Myneo Nv Product
WO2025054236A2 (en) 2023-09-06 2025-03-13 Flagship Pioneering Innovations Vii, Llc Sars-cov-2 vaccine compositions and related methods
WO2025052180A2 (en) 2023-09-07 2025-03-13 Axelyf ehf. Lipids and lipid nanoparticles
WO2025051994A1 (en) 2023-09-07 2025-03-13 Coave Therapeutics Ionizable lipid nanoparticles
WO2025054556A1 (en) 2023-09-07 2025-03-13 BioNTech SE Rna compositions for delivery of mpox antigens and related methods
WO2025057088A1 (en) 2023-09-11 2025-03-20 BioNTech SE Rna compositions for delivery of incretin agents
WO2025064475A2 (en) 2023-09-18 2025-03-27 Flagship Pioneering Innovations Vii, Llc Ionizable lipidoid compositions and therapeutic uses thereof
WO2025064660A2 (en) 2023-09-21 2025-03-27 Alnylam Pharmaceuticals, Inc. Activin a receptor type 1c (acvr1c) irna compositions and methods of use thereof
WO2025064850A1 (en) 2023-09-22 2025-03-27 BioNTech SE Rna constructs with n-terminal degrons to enhance an immune response
WO2025072383A1 (en) 2023-09-25 2025-04-03 The Broad Institute, Inc. Viral open reading frames, uses thereof, and methods of detecting the same
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025068489A1 (en) 2023-09-27 2025-04-03 Resolution Therapeutics Limited Therapeutic macrophages
WO2025072293A1 (en) 2023-09-27 2025-04-03 Scribe Therapeutics Inc. Optimized mrnas encoding casx proteins
WO2025072713A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Polymyxins for delivery of agents to the kidney
WO2025072672A2 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Slc6a19-targeting modulatory nucleic acid agents
WO2025072699A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Aminoglycosides for delivery of agents to the kidney
WO2025076031A2 (en) 2023-10-03 2025-04-10 Alnylam Pharmaceuticals, Inc. Peritoneal macrophages comprising a nanoparticle encapsulating a nucleic acid molecule and methods of use thereof
WO2025081042A1 (en) 2023-10-12 2025-04-17 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025083619A1 (en) 2023-10-18 2025-04-24 Life Edit Therapeutics, Inc. Rna-guided nucleases and acive fragments and variants thereof and methods of use
WO2025096807A2 (en) 2023-10-31 2025-05-08 Flagship Pioneering Innovations Vii, Llc Novel therapeutic dna forms
WO2025097055A2 (en) 2023-11-02 2025-05-08 The Broad Institute, Inc. Compositions and methods of use of t cells in immunotherapy
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
TW202535835A (zh) 2023-11-14 2025-09-16 美商旗艦先鋒創新有限責任(Vii)公司 可電離類脂質組成物及其治療用途
WO2025106754A1 (en) 2023-11-15 2025-05-22 BioNTech SE Coronavirus vaccine
US20250163421A1 (en) 2023-11-22 2025-05-22 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US12435338B2 (en) 2023-11-22 2025-10-07 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US20250163422A1 (en) 2023-11-22 2025-05-22 Wyvem Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US20250161347A1 (en) 2023-11-22 2025-05-22 Flagship Pioneering Innovations Vii, Llc Methods and compositions for treating non-alcoholic fatty liver disease
US12195747B1 (en) 2023-11-22 2025-01-14 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
WO2025114520A1 (en) 2023-12-01 2025-06-05 Coave Therapeutics Ionizable lipid nanoparticles
WO2025128799A1 (en) 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof
WO2025124711A1 (en) 2023-12-13 2025-06-19 BioNTech SE Glycolipid compositions
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
US20250332281A2 (en) 2023-12-15 2025-10-30 Capstan Therapeutics, Inc. Humanized anti-cd8 antibodies and uses thereof
WO2025129158A1 (en) 2023-12-15 2025-06-19 The Broad Institute, Inc. Engineered arc delivery vesicles and uses thereof
WO2025134066A1 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Ionizable lipids
WO2025132839A1 (en) 2023-12-21 2025-06-26 Glaxosmithkline Biologicals Sa Influenza virus vaccines
WO2025133105A1 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Compositions and methods
TW202525287A (zh) 2023-12-21 2025-07-01 德商拜恩技術運輸科技有限責任公司 可離子化脂質
WO2025144938A1 (en) 2023-12-26 2025-07-03 Emmune, Inc. Systems for nucleic acid transfer
WO2025140551A1 (en) 2023-12-29 2025-07-03 Neuro3 Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING NaV1.7 (SCN9A) -ASSOCIATED DISORDERS
WO2025149492A1 (en) 2024-01-08 2025-07-17 BioNTech SE Rna encoding an immune inhibitory il-1 family member
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
KR102716110B1 (ko) 2024-01-19 2024-10-15 (주) 멥스젠 다양한 크기를 갖는 지질나노입자 제조용 미세유체장치 및 이를 이용한 제조방법
US20250375499A1 (en) 2024-01-26 2025-12-11 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025166238A1 (en) 2024-01-31 2025-08-07 Orna Therapeutics, Inc. Fast-shedding polyethylene glycol lipids
WO2025166323A2 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. Crispr-related methods and compositions targeting lipoprotein (a) expression
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174908A1 (en) 2024-02-12 2025-08-21 Life Edit Therapeutics, Inc. Novel rna-guided nucleases and proteins for polymerase editing
US20250263707A1 (en) 2024-02-20 2025-08-21 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US12416020B2 (en) 2024-02-20 2025-09-16 Wyvern Pharmaceuticals Inc. Plasmid encoding a TLR3 and Fc fusion protein
WO2025184508A1 (en) 2024-03-01 2025-09-04 Acuitas Therapeutics, Inc. Materials and methods for encapsulating therapeutics in lipid nanoparticles
WO2025194138A1 (en) 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. St1cas9 compositions and methods for modulating a genome
WO2025194019A1 (en) 2024-03-14 2025-09-18 Flagship Pioneering Innovations Vii, Llc Methods for treating liver fibrosis and non-alcoholic fatty liver disease
WO2025199231A2 (en) 2024-03-20 2025-09-25 Vertex Pharmaceuticals Incorporated Mucin-5b (muc5b) targeted sirna and antisense oligonucleotides and methods of use thereof
WO2025202287A1 (en) 2024-03-26 2025-10-02 Resolution Therapeutics Limited Method of producing macrophages
WO2025202937A1 (en) 2024-03-26 2025-10-02 BioNTech SE Cancer vaccines
GB202404607D0 (en) 2024-03-29 2024-05-15 Glaxosmithkline Biologicals Sa RNA formulation
WO2025213131A1 (en) 2024-04-05 2025-10-09 BioNTech SE Rna compositions for delivery of orthopox antigens and related methods
WO2025213138A1 (en) 2024-04-05 2025-10-09 Editas Medicine, Inc. Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
WO2025219570A1 (en) 2024-04-17 2025-10-23 Johannes Kepler Universität Linz Composition for inhibiting protein translocation and methods using the same
WO2025228975A1 (en) 2024-04-29 2025-11-06 BioNTech SE Particles, compositions and methods
WO2025235862A1 (en) 2024-05-10 2025-11-13 Inndura Therapeutics Inc. A modified immune cell receptor comprising a target-binding domain and the extracellular domain of cd16a
US20250353881A1 (en) 2024-05-16 2025-11-20 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025240940A1 (en) 2024-05-17 2025-11-20 Scribe Therapeutics Inc. Compositions and methods for the targeting of apolipoprotein c3
WO2025245188A2 (en) 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Methods of treating liver steatosis and non-alcoholic fatty liver disease
WO2025245111A1 (en) 2024-05-22 2025-11-27 Flagship Pioneering Innovations Vii, Llc Immunoreceptor targeting proteins and related methods
WO2025250808A1 (en) 2024-05-29 2025-12-04 The Brigham And Women’S Hospital, Inc. Anti-crispr delivery compositions and methods
WO2025259747A2 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof
US12480129B1 (en) 2024-09-17 2025-11-25 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US12486506B1 (en) 2024-09-20 2025-12-02 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US12480123B1 (en) 2024-09-20 2025-11-25 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US12480121B1 (en) 2024-09-20 2025-11-25 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid
US12416021B1 (en) 2024-10-11 2025-09-16 Wyvern Pharmaceuticals Inc. Composition for regulating production of proteins
US12428654B1 (en) 2024-10-29 2025-09-30 Wyvern Pharmaceuticals Inc. Composition for regulating production of proteins
US12416022B1 (en) 2024-10-29 2025-09-16 Wyvern Pharmaceuticals Inc. Composition for regulating production of proteins
US12497618B1 (en) 2025-09-09 2025-12-16 Wyvern Pharmaceuticals Inc. Composition for regulating production of interfering ribonucleic acid

Family Cites Families (229)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US534484A (en) * 1895-02-19 Price and weighing scale
NL154598B (nl) 1970-11-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking.
US3901654A (en) 1971-06-21 1975-08-26 Biological Developments Receptor assays of biologically active compounds employing biologically specific receptors
US3817827A (en) 1972-03-30 1974-06-18 Scott Paper Co Soft absorbent fibrous webs containing elastomeric bonding material and formed by creping and embossing
US3935074A (en) 1973-12-17 1976-01-27 Syva Company Antibody steric hindrance immunoassay with two antibodies
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4034074A (en) 1974-09-19 1977-07-05 The Board Of Trustees Of Leland Stanford Junior University Universal reagent 2-site immunoradiometric assay using labelled anti (IgG)
US3993754A (en) 1974-10-09 1976-11-23 The United States Of America As Represented By The United States Energy Research And Development Administration Liposome-encapsulated actinomycin for cancer chemotherapy
US3984533A (en) 1975-11-13 1976-10-05 General Electric Company Electrophoretic method of detecting antigen-antibody reaction
US4086257A (en) 1976-10-12 1978-04-25 Sears Barry D Phosphatidyl quaternary ammonium compounds
US4098876A (en) 1976-10-26 1978-07-04 Corning Glass Works Reverse sandwich immunoassay
CH624011A5 (enExample) 1977-08-05 1981-07-15 Battelle Memorial Institute
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4394448A (en) * 1978-02-24 1983-07-19 Szoka Jr Francis C Method of inserting DNA into living cells
EP0032578B1 (de) * 1980-01-16 1984-07-25 Hans Georg Prof. Dr. Weder Verfahren und Dialysiereinrichtung zur Herstellung von Bilayer-Vesikeln und Verwendung der Bilayer-Vesikel
US4598051A (en) * 1980-03-12 1986-07-01 The Regents Of The University Of California Liposome conjugates and diagnostic methods therewith
US4452901A (en) 1980-03-20 1984-06-05 Ciba-Geigy Corporation Electrophoretically transferring electropherograms to nitrocellulose sheets for immuno-assays
US4376110A (en) 1980-08-04 1983-03-08 Hybritech, Incorporated Immunometric assays using monoclonal antibodies
US4522808A (en) 1980-08-15 1985-06-11 Societe Anonyme Dite: L'oreal Anti-sunburn compositions containing 2-phenyl-indole derivatives
US4522803A (en) 1983-02-04 1985-06-11 The Liposome Company, Inc. Stable plurilamellar vesicles, their preparation and use
US4588578A (en) 1983-08-08 1986-05-13 The Liposome Company, Inc. Lipid vesicles prepared in a monophase
US4515736A (en) * 1983-05-12 1985-05-07 The Regents Of The University Of California Method for encapsulating materials into liposomes
US4897355A (en) * 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5208036A (en) 1985-01-07 1993-05-04 Syntex (U.S.A.) Inc. N-(ω, (ω-1)-dialkyloxy)- and N-(ω, (ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5545412A (en) * 1985-01-07 1996-08-13 Syntex (U.S.A.) Inc. N-[1, (1-1)-dialkyloxy]-and N-[1, (1-1)-dialkenyloxy]-alk-1-yl-n,n,n-tetrasubstituted ammonium lipids and uses therefor
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US5453566A (en) 1986-03-28 1995-09-26 Calgene, Inc. Antisense regulation of gene expression in plant/cells
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
US5320906A (en) * 1986-12-15 1994-06-14 Vestar, Inc. Delivery vehicles with amphiphile-associated active ingredient
JP2539665B2 (ja) * 1988-06-20 1996-10-02 日本臓器製薬株式会社 神経疾患治療剤
CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
GB8822492D0 (en) 1988-09-24 1988-10-26 Considine J Apparatus for removing tumours from hollow organs of body
US5703055A (en) * 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
FR2645866B1 (fr) * 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5725871A (en) 1989-08-18 1998-03-10 Danbiosyst Uk Limited Drug delivery compositions comprising lysophosphoglycerolipid
AU637800B2 (en) 1989-08-31 1993-06-10 City Of Hope Chimeric dna-rna catalytic sequences
US5286634A (en) 1989-09-28 1994-02-15 Stadler Joan K Synergistic method for host cell transformation
JPH03126211A (ja) 1989-10-12 1991-05-29 Nippon Chemicon Corp 電解コンデンサ用電解液
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5225212A (en) * 1989-10-20 1993-07-06 Liposome Technology, Inc. Microreservoir liposome composition and method
US5707644A (en) 1989-11-04 1998-01-13 Danbiosyst Uk Limited Small particle compositions for intranasal drug delivery
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
JPH06171Y2 (ja) 1990-03-30 1994-01-05 日新製鋼株式会社 圧延機のパスライン切換装置
US5279833A (en) 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
AU649074B2 (en) 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
FR2674257B1 (fr) * 1991-03-20 1993-05-28 Armines Alliages a base de niobium et de titane resistant a l'oxydation a hautes temperatures.
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
JP2774417B2 (ja) 1991-08-07 1998-07-09 株式会社ディ・ディ・エス研究所 ペプチド骨格を有する分枝鎖型糖複合体及び微粒子キャリヤー
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US6831166B2 (en) 1992-10-23 2004-12-14 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5756353A (en) 1991-12-17 1998-05-26 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol-and liposome-based delivery
CA2126101A1 (en) 1991-12-17 1993-06-24 Robert J. Debs Gene therapy for cystic fibrosis transmembrane conductance regulator activity (cftr)
US5858784A (en) 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
JP2731630B2 (ja) * 1992-02-19 1998-03-25 ベイラー・カレッジ・オブ・メディスン 細胞増殖のオリゴヌクレオチドの変調
CA2135646A1 (en) 1992-05-11 1993-11-25 Kenneth G. Draper Method and reagent for inhibiting viral replication
DK0641192T3 (da) 1992-05-18 1998-03-02 Minnesota Mining & Mfg Anordning til transmucosal lægemiddelafgivelse
EP0646178A1 (en) 1992-06-04 1995-04-05 The Regents Of The University Of California expression cassette with regularoty regions functional in the mammmlian host
US5792451A (en) 1994-03-02 1998-08-11 Emisphere Technologies, Inc. Oral drug delivery compositions and methods
EP0786522A2 (en) 1992-07-17 1997-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecules for treatment of stenotic conditions
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
JP2854203B2 (ja) 1992-09-03 1999-02-03 株式会社ディ・ディ・エス研究所 リポソームの製造法
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
US5578475A (en) * 1993-07-12 1996-11-26 Life Technologies, Inc. Composition and methods for transfecting eukaryotic cells
US5426039A (en) 1993-09-08 1995-06-20 Bio-Rad Laboratories, Inc. Direct molecular cloning of primer extended DNA containing an alkane diol
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5674908A (en) 1993-12-20 1997-10-07 Life Technologies, Inc. Highly packed polycationic ammonium, sulfonium and phosphonium lipids
FR2714830B1 (fr) 1994-01-10 1996-03-22 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
US6989434B1 (en) 1994-02-11 2006-01-24 Invitrogen Corporation Reagents for intracellular delivery of macromolecules
US6075012A (en) 1994-02-11 2000-06-13 Life Technologies, Inc. Reagents for intracellular delivery of macromolecules
US5591317A (en) 1994-02-16 1997-01-07 Pitts, Jr.; M. Michael Electrostatic device for water treatment
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
US5639725A (en) 1994-04-26 1997-06-17 Children's Hospital Medical Center Corp. Angiostatin protein
WO1995035301A1 (en) 1994-06-22 1995-12-28 Megabios Corporation Cationic amphiphiles
FR2722506B1 (fr) 1994-07-13 1996-08-14 Rhone Poulenc Rorer Sa Composition contenant des acides nucleiques, preparation et utilisations
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
ATE219660T1 (de) 1994-09-30 2002-07-15 Inex Pharmaceuticals Corp Mittel zum einbringen polyanionischer materialien in zellen
US5820873A (en) * 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
AU3889595A (en) 1994-10-05 1996-05-02 Amgen, Inc. Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein
US5627159A (en) 1994-10-27 1997-05-06 Life Technologies, Inc. Enhancement of lipid cationic transfections in the presence of serum
US5783683A (en) 1995-01-10 1998-07-21 Genta Inc. Antisense oligonucleotides which reduce expression of the FGFRI gene
US5580579A (en) 1995-02-15 1996-12-03 Nano Systems L.L.C. Site-specific adhesion within the GI tract using nanoparticles stabilized by high molecular weight, linear poly (ethylene oxide) polymers
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US20030069173A1 (en) 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced transfections
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
EP0874910A4 (en) 1995-06-07 1999-04-21 Life Technologies Inc ENHANCED CATIONIC LIPID TRANSFECTION BY PEPTIDES
WO1996040726A1 (en) 1995-06-07 1996-12-19 Genta Incorporated Novel carbamate-based cationic lipids
US5976567A (en) * 1995-06-07 1999-11-02 Inex Pharmaceuticals Corp. Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US6051429A (en) 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US5705385A (en) 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
EP0840744A4 (en) 1995-06-07 1999-03-10 Genta Inc CATIONIC LIPIDS BASED ON PHOSPHONIC ACID
US5981501A (en) * 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5811406A (en) 1995-06-07 1998-09-22 Regents Of The University Of California Dry powder formulations of polynucleotide complexes
US7422902B1 (en) * 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US6339173B1 (en) 1996-07-22 2002-01-15 Promega Biosciences, Inc. Amide-based cationic lipids
US6020526A (en) 1995-07-21 2000-02-01 Genta, Incorporated Amide-based cationic lipids
US20030216335A1 (en) 2001-11-30 2003-11-20 Jennifer Lockridge Method and reagent for the modulation of female reproductive diseases and conditions
US20040142895A1 (en) 1995-10-26 2004-07-22 Sirna Therapeutics, Inc. Nucleic acid-based modulation of gene expression in the vascular endothelial growth factor pathway
US6330349B1 (en) 1995-11-30 2001-12-11 Chromavision Medical Systems, Inc. Automated method for image analysis of residual protein
US5817856A (en) 1995-12-11 1998-10-06 Yissum Research Development Company Of The Hebrew University Of Jerusalem Radiation-protective phospholipid and method
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6713069B1 (en) 1996-04-16 2004-03-30 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Compositions and methods for detecting, preventing, and treating African Hemorrhagic Fever
US20050119470A1 (en) 1996-06-06 2005-06-02 Muthiah Manoharan Conjugated oligomeric compounds and their use in gene modulation
US5841662A (en) * 1996-07-31 1998-11-24 Coburn Optical Industries, Inc. Method and apparatus for chucked work piece recognition
US6284267B1 (en) 1996-08-14 2001-09-04 Nutrimed Biotech Amphiphilic materials and liposome formulations thereof
US6111085A (en) 1996-09-13 2000-08-29 Isis Pharmaceuticals, Inc. Carbamate-derivatized nucleosides and oligonucleosides
US6174861B1 (en) 1996-10-22 2001-01-16 The Children's Medical Center Corporation Methods of inhibiting angiogenesis via increasing in vivo concentrations of endostatin protein
US6210707B1 (en) 1996-11-12 2001-04-03 The Regents Of The University Of California Methods of forming protein-linked lipidic microparticles, and compositions thereof
US5739119A (en) 1996-11-15 1998-04-14 Galli; Rachel L. Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA
US5877220A (en) 1997-03-06 1999-03-02 Genta, Incorporated Amide-based oligomeric cationic lipids
US6034135A (en) 1997-03-06 2000-03-07 Promega Biosciences, Inc. Dimeric cationic lipids
US6406705B1 (en) 1997-03-10 2002-06-18 University Of Iowa Research Foundation Use of nucleic acids containing unmethylated CpG dinucleotide as an adjuvant
EP1027033B1 (en) 1997-05-14 2009-07-22 The University Of British Columbia High efficiency encapsulation of nucleic acids in lipid vesicles
WO1998051285A2 (en) 1997-05-15 1998-11-19 Genzyme Corporation Cationic amphiphile formulations
US20030073640A1 (en) 1997-07-23 2003-04-17 Ribozyme Pharmaceuticals, Inc. Novel compositions for the delivery of negatively charged molecules
AU8428998A (en) 1997-07-24 1999-02-16 Inex Pharmaceuticals Corporation Preparation of lipid-nucleic acid particles using a solvent extraction and direct hydration method
CA2792406A1 (en) 1997-10-10 1999-04-22 Tekmira Pharmaceuticals Corporation Methods for encapsulating nucleic acids in lipid bilayers
JP2993489B2 (ja) * 1997-12-15 1999-12-20 日本電気株式会社 疑似多チャンネルステレオ再生装置
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
US6410328B1 (en) * 1998-02-03 2002-06-25 Protiva Biotherapeutics Inc. Sensitizing cells to compounds using lipid-mediated gene and compound delivery
CA2271582A1 (en) 1998-05-14 1999-11-14 Sean C. Semple Method for administration of therapeutic agents, including antisense, with repeat dosing
US6429200B1 (en) 1998-07-17 2002-08-06 Mirus Corporation Reverse micelles for delivery of nucleic acids
ATE237312T1 (de) 1998-07-20 2003-05-15 Protiva Biotherapeutics Inc In liposomen verkapselte nukleinsäurekomplexe
US6900049B2 (en) 1998-09-10 2005-05-31 Cell Genesys, Inc. Adenovirus vectors containing cell status-specific response elements and methods of use thereof
EP1129064B1 (en) 1998-11-12 2008-01-09 Invitrogen Corporation Transfection reagents
US6649780B1 (en) * 1998-12-22 2003-11-18 Valentis, Inc. Cationic lipids
ES2295018T3 (es) 1999-03-02 2008-04-16 Transave, Inc. Encapsulacion de complejos bioactivos en liposomas.
US6852334B1 (en) 1999-04-20 2005-02-08 The University Of British Columbia Cationic peg-lipids and methods of use
WO2000062813A2 (en) 1999-04-20 2000-10-26 The University Of British Columbia Cationic peg-lipids and methods of use
US6696424B1 (en) 1999-05-28 2004-02-24 Vical Incorporated Cytofectin dimers and methods of use thereof
US20010048940A1 (en) 1999-06-18 2001-12-06 Jennifer D. Tousignant Cationic amphiphile micellar complexes
HUP0202177A3 (en) 1999-07-14 2004-05-28 Alza Corp Mountain View Neutral lipopolymer and liposomal compositions containing same
IL147089A0 (en) 1999-07-15 2002-08-14 Inex Pharmaceuticals Corp Methods of preparing lipid-encapsulated therapeutic agents
CZ20021029A3 (cs) 1999-08-27 2002-08-14 Inex Pharmaceuticals Corp. Kompozice a její pouľití pro stimulaci sekrece cytokinů a indukci imunitní odpovědi
US6458382B1 (en) 1999-11-12 2002-10-01 Mirus Corporation Nucleic acid transfer complexes
AU785017B2 (en) 2000-01-10 2006-08-24 Yissum Research Development Company Of The Hebrew University Of Jerusalem Use of lipid conjugates in the treatment of disease
US20010048040A1 (en) * 2000-02-04 2001-12-06 Sheldon Paul C. Back-pressure responsive, self-reversing, control valve
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
EP1309726B2 (en) 2000-03-30 2018-10-03 Whitehead Institute For Biomedical Research Rna sequence-specific mediators of rna interference
US7189705B2 (en) 2000-04-20 2007-03-13 The University Of British Columbia Methods of enhancing SPLP-mediated transfection using endosomal membrane destabilizers
US20030072794A1 (en) 2000-06-09 2003-04-17 Teni Boulikas Encapsulation of plasmid DNA (lipogenes™) and therapeutic agents with nuclear localization signal/fusogenic peptide conjugates into targeted liposome complexes
AU2001275423B2 (en) 2000-06-09 2007-01-11 Regulon, Inc. Encapsulation of polynucleotides and drugs into targeted liposomes
US20030082103A1 (en) 2000-10-11 2003-05-01 Targesome, Inc. Targeted therapeutic lipid constructs having cell surface targets
WO2002034236A2 (en) 2000-10-25 2002-05-02 The University Of British Columbia Lipid formulations for target delivery
NZ525440A (en) 2000-10-27 2005-03-24 Invitrogen Corp Method for introducing antisense oligonucleotides into eucaryotic cells using cationic lipids
US20020081736A1 (en) 2000-11-03 2002-06-27 Conroy Susan E. Nucleic acid delivery
US20040259247A1 (en) 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
ES2307568T3 (es) 2000-12-08 2008-12-01 Coley Pharmaceutical Gmbh Acidos nucleicos de tipo cpg y metodos de uso de los mismos.
US7053150B2 (en) 2000-12-18 2006-05-30 Nektar Therapeutics Al, Corporation Segmented polymers and their conjugates
TW593427B (en) 2000-12-18 2004-06-21 Nektar Therapeutics Al Corp Synthesis of high molecular weight non-peptidic polymer derivatives
EP1386004A4 (en) * 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
US20030026831A1 (en) 2001-04-20 2003-02-06 Aparna Lakkaraju Anionic liposomes for delivery of bioactive agents
US20040142892A1 (en) * 2001-04-30 2004-07-22 The University Of British Columbia Autogene nucleic acids encoding a secretable RNA polymerase
US20030077829A1 (en) * 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
US20050282188A1 (en) 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
DE10163098B4 (de) 2001-10-12 2005-06-02 Alnylam Europe Ag Verfahren zur Hemmung der Replikation von Viren
US20040063654A1 (en) * 2001-11-02 2004-04-01 Davis Mark E. Methods and compositions for therapeutic use of RNA interference
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
WO2003070283A2 (en) 2002-02-22 2003-08-28 Klaus Strebhardt Agent for inhibiting development or progress of proliferative diseases and especially cancer diseases and pharmaceutical composition containing said agent
EP1486089A1 (en) * 2002-03-08 2004-12-15 Nokia Corporation Enhanced and device for controlling the power in an asymmetric soft handover condition
CA2752143C (en) 2002-05-15 2014-09-23 Sutter West Bay Hospitals Delivery of nucleic acid-like compounds
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
DE60326961D1 (de) 2002-07-26 2009-05-14 Novartis Vaccines & Diagnostic Modifizierte kleine irns moleküle und methoden zu deren anwendung
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
CA2513623A1 (en) 2003-01-16 2004-08-05 The Trustees Of The University Of Pennsylvania Compositions and methods for sirna inhibition of icam-1
US20040167090A1 (en) 2003-02-21 2004-08-26 Monahan Sean D. Covalent modification of RNA for in vitro and in vivo delivery
US7803781B2 (en) 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
EP1608735A4 (en) 2003-04-03 2008-11-05 Alnylam Pharmaceuticals RNAI CONJUGATES
CA2522637C (en) 2003-04-17 2014-01-21 Alnylam Pharmaceuticals, Inc. Modified irna agents
JP4782675B2 (ja) 2003-06-18 2011-09-28 イッスム・リサーチ・ディベロップメント・カンパニー・オブ・ザ・ヘブルー・ユニバーシティ・オブ・エルサレム ワクチン接種用スフィンゴイドポリアルキルアミン抱合体
KR100961267B1 (ko) * 2003-06-26 2010-06-03 엘지디스플레이 주식회사 콜레스테릭 액정 컬러필터를 가지는 투과형 액정표시장치및 그 제조 방법
ES2559828T3 (es) 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
NZ581166A (en) 2003-09-15 2011-06-30 Protiva Biotherapeutics Inc Polyethyleneglycol-modified lipid compounds and uses thereof
WO2005121348A1 (en) 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
AU2005259799A1 (en) * 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory siRNA molecules and uses therefor
JP2008512500A (ja) 2004-09-10 2008-04-24 ソマジェニックス インコーポレーティッド ウィルス遺伝子発現を効率的に阻害する低分子干渉rnaおよびその使用方法
EP1828219A4 (en) 2004-11-17 2008-07-23 Protiva Biotherapeutics Inc ARNSI SILENCE OF APOLIPOPROTEIN B
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
JP5042863B2 (ja) 2005-02-14 2012-10-03 サーナ・セラピューティクス・インコーポレイテッド 生物学的に活性な分子をデリバリーするための脂質ナノ粒子系組成物および方法
US20060228406A1 (en) 2005-03-17 2006-10-12 Invitrogen Corporation Transfection reagent for non-adherent suspension cells
AU2006274413B2 (en) * 2005-07-27 2013-01-10 Arbutus Biopharma Corporation Systems and methods for manufacturing liposomes
WO2007048046A2 (en) * 2005-10-20 2007-04-26 Protiva Biotherapeutics, Inc. Sirna silencing of filovirus gene expression
CN101346393B (zh) * 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
US20070218122A1 (en) 2005-11-18 2007-09-20 Protiva Biotherapeutics, Inc. siRNA silencing of influenza virus gene expression
EP1981543A1 (en) * 2006-01-23 2008-10-22 Abbott Laboratories Chemically modified polycation polymer for sirna delivery
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
LT2494993T (lt) 2007-05-04 2018-12-27 Marina Biotech, Inc. Aminorūgščių lipidai ir jų panaudojimas
JP5697988B2 (ja) * 2007-12-27 2015-04-08 プロチバ バイオセラピューティクス インコーポレイティッド 干渉rnaを使用したポロ様キナーゼ発現のサイレンシング方法
US20110117125A1 (en) 2008-01-02 2011-05-19 Tekmira Pharmaceuticals Corporation Compositions and methods for the delivery of nucleic acids
CN105267233B (zh) 2008-03-05 2019-07-26 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因表达的组合物和方法
JP2011516094A (ja) 2008-04-15 2011-05-26 プロチバ バイオセラピューティクス インコーポレイティッド 干渉rnaを用いたcsn5遺伝子発現のサイレンシング方法
PL2279254T3 (pl) 2008-04-15 2017-11-30 Protiva Biotherapeutics Inc. Nowe preparaty lipidowe do dostarczania kwasów nukleinowych
WO2010042877A1 (en) 2008-10-09 2010-04-15 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
HUE026604T2 (hu) 2008-10-20 2016-06-28 Alnylam Pharmaceuticals Inc Transztiretin-expresszió gátlására szolgáló készítmények és eljárások
CA2751342C (en) 2009-01-29 2019-05-07 Alnylam Pharmaceuticals, Inc. Lipid formulations comprising cationic lipid and a targeting lipid comprising n-acetyl galactosamine for delivery of nucleic acid
CA2754043A1 (en) 2009-03-12 2010-09-16 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
SG10201912450XA (en) 2009-06-10 2020-03-30 Arbutus Biopharma Corp Improved lipid formulation
US9051567B2 (en) * 2009-06-15 2015-06-09 Tekmira Pharmaceuticals Corporation Methods for increasing efficacy of lipid formulated siRNA
WO2011000108A1 (en) * 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein b
WO2011000106A1 (en) * 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Improved cationic lipids and methods for the delivery of therapeutic agents
JP5766188B2 (ja) 2009-07-01 2015-08-19 プロチバ バイオセラピューティクス インコーポレイティッド 固形腫瘍に治療剤を送達するための脂質製剤
US9018187B2 (en) * 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US8716464B2 (en) * 2009-07-20 2014-05-06 Thomas W. Geisbert Compositions and methods for silencing Ebola virus gene expression
US8455455B1 (en) * 2010-03-31 2013-06-04 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes involved in hemorrhagic fever
WO2011141703A1 (en) * 2010-05-12 2011-11-17 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
WO2011141704A1 (en) * 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
US8466122B2 (en) * 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
EP2640700B1 (en) 2010-11-15 2018-10-31 Life Technologies Corporation Amine-containing transfection reagents and methods for making and using same
US9126966B2 (en) * 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
AU2012318249B2 (en) 2011-10-05 2016-04-21 Protiva Biotherapeutics Inc. Compositions and methods for silencing aldehyde dehydrogenase
DE12858350T1 (de) 2011-12-16 2021-10-07 Modernatx, Inc. Modifizierte mrna zusammensetzungen
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
US10421208B2 (en) 2013-03-12 2019-09-24 Sunpower Corporation Method and device for cleaving wafers
WO2016033326A2 (en) 2014-08-29 2016-03-03 Alnylam Pharmaceuticals, Inc. Methods of treating transthyretin (ttr) mediated amyloidosis
CA2963271A1 (en) * 2014-10-02 2016-04-07 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis b virus gene expression
US20200315967A1 (en) 2016-06-24 2020-10-08 Modernatx, Inc. Lipid nanoparticles
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS

Similar Documents

Publication Publication Date Title
JP2011516586A5 (enExample)
CA2532228C (en) Lipid encapsulated interfering rna
JP6409011B2 (ja) 活性剤の送達のための生分解性脂質
JP5697988B2 (ja) 干渉rnaを使用したポロ様キナーゼ発現のサイレンシング方法
JP2011516094A5 (enExample)
JP2012531469A5 (enExample)
JP2017536092A5 (enExample)
JP2011507534A5 (enExample)
JP2014530602A5 (enExample)
JP2015509505A5 (enExample)
CA2979998A1 (en) Compositions and methods for treating hypertriglyceridemia
CN108024960A (zh) 含有阴离子药物的药物组合物及其制备方法
RU2017114964A (ru) Композиции и способы для подавления экспрессии гена вируса гепатита в
CN105142614A (zh) 用于配制阴离子试剂的方法
ES2901102T3 (es) Método para preparar una micela polimérica que contiene fármaco aniónico
JP2011529912A5 (enExample)
JPWO2023044343A5 (enExample)
JP2012532839A5 (enExample)
JP2010533178A5 (enExample)
JP2022087209A (ja) B型肝炎を治療するための治療用組成物及び方法
JP2020512293A (ja) インフュージョンリアクションを改善するための方法
JPWO2023044333A5 (enExample)
CN115385820B (zh) 阳离子脂质及其应用
JPWO2020097520A5 (enExample)
JP2025504405A (ja) 生分解性エステル結合を含むイオン化可能な脂質およびこれを含む脂質ナノ粒子