JP7114485B2 - ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド - Google Patents

ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド Download PDF

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JP7114485B2
JP7114485B2 JP2018560595A JP2018560595A JP7114485B2 JP 7114485 B2 JP7114485 B2 JP 7114485B2 JP 2018560595 A JP2018560595 A JP 2018560595A JP 2018560595 A JP2018560595 A JP 2018560595A JP 7114485 B2 JP7114485 B2 JP 7114485B2
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gla
alkyl
alkenyl
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mrna
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JP2019516719A (ja
JP2019516719A5 (enExample
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パオロ マルティーニ，
スティーブン ホッジ，
ケリー ベネナート，
ウラジミール プレスニャク，
イアン マクファディン，
エララヘワーゲ サティヤジト クマーラシンヘ，
スーリン チュー，
リン タン グエイ，
ステーシ サブニス，
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ModernaTx Inc
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    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
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    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

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JP2018560595A 2016-05-18 2017-05-18 ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド Active JP7114485B2 (ja)

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JP2022119527A JP2022136230A (ja) 2016-05-18 2022-07-27 ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド

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US201662338354P 2016-05-18 2016-05-18
US62/338,354 2016-05-18
PCT/US2017/033398 WO2017201328A1 (en) 2016-05-18 2017-05-18 POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE

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JP2022119527A Pending JP2022136230A (ja) 2016-05-18 2022-07-27 ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド

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Cited By (1)

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JP2022031359A (ja) * 2015-12-10 2022-02-18 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

Families Citing this family (90)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
CN105939767B (zh) 2014-01-08 2018-04-06 弗洛设计声能学公司 具有双声电泳腔的声电泳装置
HRP20220070T1 (hr) 2014-04-23 2022-04-01 Modernatx, Inc. Cjepiva nukleinske kiseline
CA2970638A1 (en) 2014-12-22 2016-06-30 Codexis, Inc. Human alpha-galactosidase variants
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
MA42502A (fr) 2015-07-21 2018-05-30 Modernatx Inc Vaccins contre une maladie infectieuse
EP3328394A4 (en) 2015-07-30 2019-03-13 ModernaTX, Inc. CONCATEMEE peptide epitope RNAs
WO2017031232A1 (en) 2015-08-17 2017-02-23 Modernatx, Inc. Methods for preparing particles and related compositions
HRP20220156T1 (hr) 2015-09-17 2022-04-15 Modernatx, Inc. Spojevi i pripravci za unutarstaničnu isporuku terapeutskih sredstava
EP3364949A4 (en) 2015-10-22 2019-07-31 ModernaTX, Inc. CANCER VACCINES
WO2017070624A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Tropical disease vaccines
SG11201803360UA (en) 2015-10-22 2018-05-30 Modernatx Inc Nucleic acid vaccines for varicella zoster virus (vzv)
EP3364981A4 (en) 2015-10-22 2019-08-07 ModernaTX, Inc. VACCINE AGAINST THE HUMAN CYTOMEGALOVIRUS
DE20164728T1 (de) 2015-10-22 2021-09-30 Modernatx, Inc. Impfstoffe gegen atemwegsvirus
RS63051B1 (sr) 2015-12-22 2022-04-29 Modernatx Inc Jedinjenja i kompozicije za intracelularnu isporuku agenasa
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
JP7114485B2 (ja) 2016-05-18 2022-08-08 モデルナティエックス インコーポレイテッド ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド
US12128113B2 (en) 2016-05-18 2024-10-29 Modernatx, Inc. Polynucleotides encoding JAGGED1 for the treatment of Alagille syndrome
SG11201901941YA (en) 2016-09-14 2019-04-29 Modernatx Inc High purity rna compositions and methods for preparation thereof
EP3528821A4 (en) 2016-10-21 2020-07-01 ModernaTX, Inc. HUMAN CYTOMEGALOVIRUS VACCINE
MA46766A (fr) 2016-11-11 2019-09-18 Modernatx Inc Vaccin antigrippal
EP3551193A4 (en) 2016-12-08 2020-08-19 Modernatx, Inc. NUCLEIC ACID VACCINES AGAINST RESPIRATORY VIRUSES
EP3555289A1 (en) 2016-12-13 2019-10-23 ModernaTX, Inc. Rna affinity purification
JP7332478B2 (ja) 2017-03-15 2023-08-23 モデルナティエックス インコーポレイテッド 脂質ナノ粒子製剤
WO2018170270A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Varicella zoster virus (vzv) vaccine
WO2018170256A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Herpes simplex virus vaccine
US11576961B2 (en) 2017-03-15 2023-02-14 Modernatx, Inc. Broad spectrum influenza virus vaccine
WO2018170322A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Crystal forms of amino lipids
WO2018170260A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Respiratory syncytial virus vaccine
RS63953B1 (sr) 2017-03-15 2023-02-28 Modernatx Inc Jedinjenje i kompozicije za intracelularnu isporuku terapeutskih sredstava
WO2018170347A1 (en) 2017-03-17 2018-09-20 Modernatx, Inc. Zoonotic disease rna vaccines
EP3601580A4 (en) * 2017-03-23 2021-01-06 Medicago Inc. NOROVIRUS FUSION PROTEINS AND VLPS WITH NOROVIRUS FUSION PROTEINS
MX2019011215A (es) 2017-03-24 2020-02-12 Curevac Ag Ácidos nucleicos que codifican proteínas asociadas a crispr y usos de los mismos.
MA48047A (fr) 2017-04-05 2020-02-12 Modernatx Inc Réduction ou élimination de réponses immunitaires à des protéines thérapeutiques administrées par voie non intraveineuse, par exemple par voie sous-cutanée
EP3638678B1 (en) 2017-06-14 2025-12-03 ModernaTX, Inc. Compounds and compositions for intracellular delivery of agents
MA49421A (fr) 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
WO2019036685A1 (en) 2017-08-18 2019-02-21 Modernatx, Inc. METHODS FOR HPLC ANALYSIS
WO2019036682A1 (en) 2017-08-18 2019-02-21 Modernatx, Inc. RNA VARIANTS POLYMERASE
EP3668977A4 (en) 2017-08-18 2021-04-21 Modernatx, Inc. HPLC ANALYTICAL PROCESSES
MX2020002348A (es) * 2017-08-31 2020-10-08 Modernatx Inc Métodos de elaboración de nanopartículas lipídicas.
EP3681514A4 (en) 2017-09-14 2021-07-14 ModernaTX, Inc. RNA VACZINE AGAINST ZIKA VIRUS
CN114900773A (zh) 2017-12-14 2022-08-12 弗洛设计声能学公司 声泳系统及其操作方法、控制声换能器及声学系统的方法
CN111712468A (zh) 2017-12-22 2020-09-25 北卡罗莱纳州立大学 聚合物荧光团、包含其的组合物及制备和使用其的方法
US11911453B2 (en) 2018-01-29 2024-02-27 Modernatx, Inc. RSV RNA vaccines
US20190314291A1 (en) * 2018-01-30 2019-10-17 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells
WO2019169333A1 (en) 2018-03-02 2019-09-06 Sigilon Therapeutics, Inc. Afibrotic compounds, devices, and uses thereof
EP3759154A1 (en) 2018-03-02 2021-01-06 Sigilon Therapeutics, Inc. Biocompatible hydrogel capsules and process for preparing same
CN112203646A (zh) 2018-04-04 2021-01-08 西吉隆医疗股份有限公司 可植入颗粒和相关方法
US12151029B2 (en) 2018-09-19 2024-11-26 Modernatx, Inc. PEG lipids and uses thereof
CN112996854B (zh) 2018-09-19 2024-08-30 摩登纳特斯有限公司 高纯度peg脂质和其用途
EP3853202A1 (en) 2018-09-19 2021-07-28 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
MY207075A (en) 2018-12-20 2025-01-28 Codexis Inc Human alpha-galactosidase variants
CN113423419A (zh) * 2019-01-04 2021-09-21 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
CN113939282A (zh) 2019-01-31 2022-01-14 摩登纳特斯有限公司 制备脂质纳米颗粒的方法
US11351242B1 (en) 2019-02-12 2022-06-07 Modernatx, Inc. HMPV/hPIV3 mRNA vaccine composition
US11851694B1 (en) 2019-02-20 2023-12-26 Modernatx, Inc. High fidelity in vitro transcription
MX2021010075A (es) 2019-02-20 2021-12-10 Modernatx Inc Variantes de arn polimerasa para la formacion de casquetes cotranscripcionales.
MX2021011031A (es) 2019-03-11 2021-12-10 Modernatx Inc Proceso de transcripción in vitro en lotes alimentados.
MA55321A (fr) 2019-03-15 2022-01-19 Modernatx Inc Vaccins à base d'arn contre le vih
US12649930B2 (en) 2019-03-27 2026-06-09 Sigilon Therapeutics, Inc. Compositions, devices, and methods for treating Fabry disease
EP3965830A1 (en) * 2019-05-07 2022-03-16 ModernaTX, Inc. Differentially expressed immune cell micrornas for regulation of protein expression
WO2021026358A1 (en) * 2019-08-07 2021-02-11 Moderna TX, Inc. Compositions and methods for enhanced delivery of agents
AU2020351220A1 (en) 2019-09-19 2022-04-07 Modernatx, Inc. Carbonate containing lipid compounds and compositions for intracellular delivery of therapeutic agents
EP4031524A1 (en) 2019-09-19 2022-07-27 ModernaTX, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
TW202146648A (zh) * 2020-02-28 2021-12-16 美商克迪科思股份有限公司 人類α-半乳糖苷酶變異體
WO2021183895A1 (en) * 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
WO2021204179A1 (en) 2020-04-09 2021-10-14 Suzhou Abogen Biosciences Co., Ltd. Nucleic acid vaccines for coronavirus
TWI899203B (zh) 2020-04-09 2025-10-01 大陸商蘇州艾博生物科技有限公司 脂質奈米粒子組成物
KR102314642B1 (ko) * 2020-04-17 2021-10-20 재단법인 아산사회복지재단 파브리 병 진단용 바이오 마커 및 이의 용도
CN118490818A (zh) 2020-04-22 2024-08-16 生物技术欧洲股份公司 冠状病毒疫苗
CA3175905A1 (en) * 2020-04-27 2021-11-04 David H. Kirn Codon optimized gla genes and uses thereof
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
MX2023004371A (es) 2020-10-14 2023-07-26 George Mason Res Foundation Inc Lípidos ionizables, y métodos de fabricación y uso de los mismos.
WO2022150717A1 (en) 2021-01-11 2022-07-14 Modernatx, Inc. Seasonal rna influenza virus vaccines
PE20240915A1 (es) * 2021-01-14 2024-04-30 Spark Therapeutics Inc Composiciones y metodos para tratar la enfermedad de fabry
NL2027654B1 (en) * 2021-02-08 2022-09-09 Nutcracker Therapeutics Inc Methods for manufacturing a syntetic template
EP4326227A1 (en) 2021-04-23 2024-02-28 ModernaTX, Inc. Stabilized formulations
US20220363937A1 (en) 2021-05-14 2022-11-17 Armstrong World Industries, Inc. Stabilization of antimicrobial coatings
US12186387B2 (en) 2021-11-29 2025-01-07 BioNTech SE Coronavirus vaccine
US20230340077A1 (en) * 2021-12-01 2023-10-26 Luyef Biotechnologies Inc. Production of myoglobin from trichoderma using a feeding media
US12529047B1 (en) 2021-12-21 2026-01-20 Modernatx, Inc. mRNA quantification methods
US20250099392A1 (en) * 2022-01-20 2025-03-27 Ohio State Innovation Foundation Compositions comprising lipid compounds and methods of making and use thereof
CN114672501B (zh) * 2022-05-27 2022-08-23 珠海丽凡达生物技术有限公司 一种mRNA、药物组合物及其应用
US11878055B1 (en) 2022-06-26 2024-01-23 BioNTech SE Coronavirus vaccine
WO2026085234A1 (en) * 2024-10-18 2026-04-23 Modernatx, Inc. 3' untranslated region sequences for extending messenger ribonucleic acids half-life
CN121293183B (zh) * 2025-12-15 2026-03-24 北京悦康科创医药科技股份有限公司 含有苯并噻吩结构用于激活sting通路的佐剂脂质及其组合物

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013016058A1 (en) 2011-07-22 2013-01-31 Merck Sharp & Dohme Corp. Novel bis-nitrogen containing cationic lipids for oligonucleotide delivery
JP2014523411A (ja) 2011-06-08 2014-09-11 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド ｍＲＮＡ送達のための脂質ナノ粒子組成物および方法
WO2014164253A1 (en) 2013-03-09 2014-10-09 Moderna Therapeutics, Inc. Heterologous untranslated regions for mrna
WO2015199952A1 (en) 2014-06-25 2015-12-30 Acuitas Therapeutics Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids

Family Cites Families (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5082767A (en) 1989-02-27 1992-01-21 Hatfield G Wesley Codon pair utilization
US20090042283A1 (en) 1998-09-29 2009-02-12 Shire Human Genetic Therapies, Inc., A Delaware Corporation Optimized messenger rna
AU6270499A (en) 1998-09-29 2000-04-17 Phylos, Inc. Synthesis of codon randomized nucleic acids
DK1325138T3 (da) 2000-10-11 2013-10-21 Shire Human Genetic Therapies Optimeret messenger rna
ATE291925T1 (de) 2001-06-05 2005-04-15 Curevac Gmbh Stabilisierte mrna mit erhöhtem g/c-gehalt und optimierter codon usage für die gentherapie
EP2428570B1 (en) 2001-09-28 2018-05-23 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Microrna molecules
EP1490494A1 (en) 2002-04-01 2004-12-29 Walter Reed Army Institute of Research Method of designing synthetic nucleic acid sequences for optimal protein expression in a host cell
DE10260805A1 (de) 2002-12-23 2004-07-22 Geneart Gmbh Verfahren und Vorrichtung zum Optimieren einer Nucleotidsequenz zur Expression eines Proteins
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
US8101385B2 (en) 2005-06-30 2012-01-24 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
AU2006265896B2 (en) 2005-06-30 2012-05-31 Archemix Corp. Materials and methods for the generation of fully 2'-modified nucleic acid transcripts
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
US20080046192A1 (en) 2006-08-16 2008-02-21 Richard Lathrop Polypepetide-encoding nucleotide sequences with refined translational kinetics and methods of making same
AU2007337810B2 (en) 2006-12-22 2014-02-13 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
WO2009030254A1 (en) 2007-09-04 2009-03-12 Curevac Gmbh Complexes of rna and cationic peptides for transfection and for immunostimulation
DK2205618T3 (en) 2007-09-26 2017-02-20 Intrexon Corp SYNTHETIC 5 'NON-TRANSLATED REGIONS, EXPRESSION VECTORS AND PROCEDURES FOR INCREASING TRANSGENIC EXPRESSION
SG10201408164XA (en) 2007-12-11 2015-01-29 Scripps Research Inst Compositions and methods related to mrna translational enhancer elements
PL215513B1 (pl) 2008-06-06 2013-12-31 Univ Warszawski Nowe boranofosforanowe analogi dinukleotydów, ich zastosowanie, czasteczka RNA, sposób otrzymywania RNA oraz sposób otrzymywania peptydów lub bialka
US8126653B2 (en) 2008-07-31 2012-02-28 Dna Twopointo, Inc. Synthetic nucleic acids for expression of encoded proteins
US8401798B2 (en) 2008-06-06 2013-03-19 Dna Twopointo, Inc. Systems and methods for constructing frequency lookup tables for expression systems
US7561973B1 (en) 2008-07-31 2009-07-14 Dna Twopointo, Inc. Methods for determining properties that affect an expression property value of polynucleotides in an expression system
KR20220038506A (ko) 2009-06-10 2022-03-28 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
US20110082055A1 (en) 2009-09-18 2011-04-07 Codexis, Inc. Reduced codon mutagenesis
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
US9999673B2 (en) 2011-01-11 2018-06-19 Alnylam Pharmaceuticals, Inc. PEGylated lipids and their use for drug delivery
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
EP4074693A1 (en) 2011-06-08 2022-10-19 Translate Bio, Inc. Cleavable lipids
WO2013039857A1 (en) 2011-09-12 2013-03-21 modeRNA Therapeutics Engineered nucleic acids and methods of use thereof
PT3682905T (pt) 2011-10-03 2022-04-07 Modernatx Inc Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações
SG10201604810UA (en) 2012-02-07 2016-08-30 Global Bio Therapeutics Inc Compartmentalized method of nucleic acid delivery and compositions and uses thereof
WO2013120497A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
BR112014024131A2 (pt) 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
US9512456B2 (en) 2012-08-14 2016-12-06 Modernatx, Inc. Enzymes and polymerases for the synthesis of RNA
WO2014093924A1 (en) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
US20150366997A1 (en) 2012-12-07 2015-12-24 Shire Human Genetics Therapies, Inc. COMPOSITIONS AND METHODS FOR mRNA DELIVERY
EP3434774A1 (en) * 2013-01-17 2019-01-30 ModernaTX, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
US10266559B2 (en) 2013-03-14 2019-04-23 Translate Bio, Inc. Ribonucleic acids with 4′-thio-modified nucleotides and related methods
CN105143456A (zh) 2013-03-15 2015-12-09 不列颠哥伦比亚大学 用于转染的脂质纳米粒子和相关方法
ES2967701T3 (es) 2013-03-15 2024-05-03 Translate Bio Inc Mejora sinergística de la entrega de ácidos nucleicos mediante formulaciones mezcladas
US10023626B2 (en) * 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
TWI793159B (zh) * 2013-10-23 2023-02-21 美商健臻公司 重組醣蛋白及其用途
WO2015130584A2 (en) 2014-02-25 2015-09-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
HRP20220070T1 (hr) * 2014-04-23 2022-04-01 Modernatx, Inc. Cjepiva nukleinske kiseline
WO2016118724A1 (en) * 2015-01-21 2016-07-28 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
EP3303377B1 (en) 2015-06-05 2022-11-23 Dana-Farber Cancer Institute, Inc. Compositions and methods for transient gene therapy with enhanced stability
HRP20220156T1 (hr) * 2015-09-17 2022-04-15 Modernatx, Inc. Spojevi i pripravci za unutarstaničnu isporuku terapeutskih sredstava
WO2017153936A1 (en) 2016-03-10 2017-09-14 Novartis Ag Chemically modified messenger rna's
WO2017191274A2 (en) 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
JP7114485B2 (ja) 2016-05-18 2022-08-08 モデルナティエックス インコーポレイテッド ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド
JP7560946B2 (ja) 2016-11-10 2024-10-03 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａの皮下送達

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014523411A (ja) 2011-06-08 2014-09-11 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド ｍＲＮＡ送達のための脂質ナノ粒子組成物および方法
US20140294938A1 (en) 2011-06-08 2014-10-02 Shire Human Genetic Therapies, Inc. Mrna therapy for fabry disease
WO2013016058A1 (en) 2011-07-22 2013-01-31 Merck Sharp & Dohme Corp. Novel bis-nitrogen containing cationic lipids for oligonucleotide delivery
WO2014164253A1 (en) 2013-03-09 2014-10-09 Moderna Therapeutics, Inc. Heterologous untranslated regions for mrna
WO2015199952A1 (en) 2014-06-25 2015-12-30 Acuitas Therapeutics Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
BIODRUGS，2013年06月，VOL:27, NR:3，PAGE(S):237 - 246

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022031359A (ja) * 2015-12-10 2022-02-18 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
JP7412840B2 (ja) 2015-12-10 2024-01-15 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

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