DK2718269T3 - Spaltelige lipider - Google Patents

Spaltelige lipider Download PDF

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Publication number
DK2718269T3
DK2718269T3 DK12728004.8T DK12728004T DK2718269T3 DK 2718269 T3 DK2718269 T3 DK 2718269T3 DK 12728004 T DK12728004 T DK 12728004T DK 2718269 T3 DK2718269 T3 DK 2718269T3
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lipid
mrna
polynucleotides
lipid nanoparticle
compositions
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DK12728004.8T
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English (en)
Inventor
Michael Heartlein
Braydon Charles Guild
Frank Derosa
Jerry Chi Zhang
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Translate Bio Inc
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    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
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    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J43/00—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton
    • C07J43/003—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton not condensed
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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Claims (17)

1. Lipidnanopartikel omfattende en forbindelse med strukturen:
hvor Ri er valgt fra gruppen bestående af imidazol, guanidinium, imin, enamin, amino, en eventuelt-substitueret alkylamino og pyridyl; R3 og R4 er hver uafhængigt et eventuelt substitueret, umættet C6-C20 alkyl, hvor disse eventuelle substituenter er en eller flere af alkyl, halo, alkoxyl, hydroxy, amino, aryl, ether, ester eller amid; og n er nul eller et hvilket som helst positivt heltal; endvidere omfattende et eller flere polynukleotider indkapslet inden i lipidnanopartiklen, eventuelt hvor et eller flere af polynukleotiderne omfatter en kemisk modificering.
2. Lipidnanopartikel ifølge krav 1, hvor R3 og R4 hver er (i) et eventuelt substitueret, poly-umættet C6-C20 alkyl; (ii) et eventuelt substitueret, poly-umættet Cis alkyl; eller (iii) et ikke-substitueret, poly-umættet Cis alkyl.
3. Lipidnanopartikel ifølge krav 1 eller 2, hvor Ri er amino, imidazol eller guanidinium.
4. Lipidnanopartikel ifølge krav 3, hvor R3 og R4 hver er et ikke-substitueret, poly-umættet Cie-alkyl, og n er 1.
5. Lipidnanopartikel ifølge krav 1, hvor forbindelsens struktur er:
6. Lipidnanopartikel ifølge krav 1, hvor forbindelsens struktur er:
7. Lipidnanopartikel ifølge et hvilket som helst af kravene 1-6, endvidere omfattende en eller flere forbindelser valgt fra gruppen bestående af et kationisk lipid, et PEG-modificeret lipid, et ikke-kationisk lipid og et hjælper-lipid.
8. Lipidnanopartikel ifølge krav 7, hvor det kationiske lipid er valgt fra gruppen bestående af C12-200, DOTAP (l,2-dioleyl-3-trimethylammoniumpropan), DODAP (l,2-dioleyl-3-dimethylammoniumpropan), DOTMA (l,2-di-0-octadecenyl-3-trimethylammoniumpropan), DLinDMA (l,2-dilinoleyloxy-/V,/V-dimethylpropylamin), DLinKC2-DMA (l,2-dilinoleyl-4-(2-dimethylaminoethyl)-[l,3]-dioxolan), HGT4003 (2-((2,3-Bis((9Z, 12Z)-octadeca-9,12-dien-l-yloxy)propyl)disulfanyl)-N,N-dimethylethanamin), HGT5001 ((15Z,18Z)-N,N-dimethyl-6-((9Z,12Z)-octadeca-9,12-dien-l-yl)tetracosa-4,15,18-trien-l-amin), og ICE (imidazolcholesterolester).
9. Lipidnanopartikel ifølge krav 7 eller 8, hvor nanopartiklen omfatter et eller flere PEG-modificerede lipider, eventuelt hvor det ene eller flere PEG-modificerede lipider omfatter en poly(ethylen)glycolkæde med op til 5kDa i længden kovalent bundet til et lipid omfattende en eller flere alkylkæder med C6-C20 i længden.
10. Lipidnanopartikel ifølge et hvilket som helst af de foregående krav, hvor lipidnanopartiklen omfatter et kationisk lipid, et PEG-modificeret lipid, et ikke-kationisk lipid og cholesterol.
11. Lipidnanopartikel ifølge et hvilket som helst af de foregående krav, hvor det ene eller flere polynukleotider er valgt fra gruppen bestående af et antisense oligonukleotid, siRNA, miRNA, snRNA, snoRNA og kombinationer deraf.
12. Lipidnanopartikel ifølge et hvilket som helst af kravene 1-11, hvor det ene eller flere polynukleotider omfatter et eller flere af LNA, DNA eller RNA.
13. Lipidnanopartikel ifølge krav 13, hvor RNA'et koder for et enzym, eventuelt, hvor enzymet er valgt fra gruppen bestående af cystisk fibrose transmembran konduktans regulator (CFTR), agalsidase alfa, alpha-L-iduronidase, iduronat-2-sulfatase, N-acetylglucosamin-l-phosphat-transferase, N-acetylglucosaminidase, alpha-glucosaminide-acetyltransferase, N-acetylglucosamin-6-sulfatase, N-acetylgalactosamin-4-sulfatase, beta-glucosidase, galactose-6-sulfat-sulfatase, beta-galactosidase, beta-glucuronidase, glucocerebrosidase, heparan sulfamidase, hyaluronidase, galactocerebrosidase, ornithin transcarbamylase (OTC), carbamoyl-phosphat-synthetase 1 (CPS1), argininosuccinat-synthetase 1 (ASSI), argininosuccinat-lyase (ASL), og arginase 1 (ARGI).
14. Farmaceutisk sammensætning omfattende lipidnanopartiklen ifølge et hvilket som helst af de foregående krav.
15. Farmaceutisk sammensætning ifølge krav 14 omfattende lipidnanopartiklen i lyofiliseret form.
16. Farmaceutisk sammensætning ifølge krav 15, hvor sammensætningen endvidere omfatter en eller flere lyobeskyttelsesmidler.
17. Farmaceutisk sammensætning ifølge et hvilket som helst af kravene 14-16 til anvendelse som et medikament, især, til anvendelse til behandling af en sygdom forbundet med afvigende ekspression af et gen eller en nukleinsyre hos et individ.
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Families Citing this family (266)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010053572A2 (en) 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
PT2506857T (pt) 2009-12-01 2018-05-14 Translate Bio Inc Entrega de arnm para o acréscimo de proteínas e enzimas em doenças genéticas humanas
CA3169291A1 (en) 2010-07-06 2012-01-12 Glaxosmithkline Biologicals Sa Immunisation of large mammals with low doses of rna
DK3243526T3 (da) 2010-07-06 2020-02-17 Glaxosmithkline Biologicals Sa Levering af rna til at udløse flere immunsignalveje
EP2590626B1 (en) 2010-07-06 2015-10-28 GlaxoSmithKline Biologicals SA Liposomes with lipids having an advantageous pka-value for rna delivery
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2609135A4 (en) 2010-08-26 2015-05-20 Massachusetts Inst Technology POLY (BETA-AMINO ALCOHOLS), THEIR PREPARATION AND USES THEREOF
HRP20221533T1 (hr) 2010-08-31 2023-02-17 Glaxosmithkline Biologicals Sa Pegilirani liposomi za isporuku rna koja kodira imunogen
DK3590949T3 (da) 2010-10-01 2022-07-11 Modernatx Inc Ribonukleinsyrer indeholdende n1-methyl-pseudouracil og anvendelse heraf
MX363307B (es) 2010-10-11 2019-03-20 Novartis Ag Star Plataformas para suministro de antigenos.
CA2831392C (en) 2011-03-28 2020-04-28 Massachusetts Institute Of Technology Conjugated lipomers and uses thereof
US8710200B2 (en) 2011-03-31 2014-04-29 Moderna Therapeutics, Inc. Engineered nucleic acids encoding a modified erythropoietin and their expression
AU2012267578B2 (en) 2011-06-08 2017-04-20 Translate Bio, Inc. Cleavable lipids
CA3107288A1 (en) 2011-06-08 2012-12-13 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mrna delivery
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
RU2648950C2 (ru) 2011-10-03 2018-04-02 Модерна Терапьютикс, Инк. Модифицированные нуклеозиды, нуклеотиды и нуклеиновые кислоты и их применение
CN107522664B (zh) 2011-10-27 2021-03-16 麻省理工学院 能够形成药物包封微球的在n末端上官能化的氨基酸衍生物
US20130156849A1 (en) 2011-12-16 2013-06-20 modeRNA Therapeutics Modified nucleoside, nucleotide, and nucleic acid compositions
CA2868030C (en) 2012-03-29 2021-05-25 Shire Human Genetic Therapies, Inc. Lipid-derived neutral nanoparticles
CA2868034C (en) 2012-03-29 2021-07-27 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
WO2013151667A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9303079B2 (en) 2012-04-02 2016-04-05 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
AU2013243949A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
EP2882706A1 (en) 2012-08-13 2015-06-17 Massachusetts Institute of Technology Amine-containing lipidoids and uses thereof
EP2885419A4 (en) 2012-08-14 2016-05-25 Moderna Therapeutics Inc ENZYMES AND POLYMERASES FOR RNA SYNTHESIS
RS63237B1 (sr) 2012-11-26 2022-06-30 Modernatx Inc Terminalno modifikovana rnk
WO2014089486A1 (en) * 2012-12-07 2014-06-12 Shire Human Genetic Therapies, Inc. Lipidic nanoparticles for mrna delivering
US20160024181A1 (en) 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
WO2014152940A1 (en) 2013-03-14 2014-09-25 Shire Human Genetic Therapies, Inc. Mrna therapeutic compositions and use to treat diseases and disorders
EA037922B1 (ru) 2013-03-14 2021-06-07 Шир Хьюман Дженетик Терапис, Инк. мРНК CFTR И КОМПОЗИЦИИ ДЛЯ ЛЕЧЕНИЯ МУКОВИСЦИДОЗА У МЛЕКОПИТАЮЩЕГО
EP2970955B1 (en) 2013-03-14 2018-11-14 Translate Bio, Inc. Methods for purification of messenger rna
HUE055044T2 (hu) 2013-03-14 2021-10-28 Translate Bio Inc MRNS-kódolt ellenanyagok bejuttatására szolgáló eljárások és készítmények
EP3750903A1 (en) 2013-03-14 2020-12-16 Translate Bio, Inc. Ribonucleic acids with 4'-thio-modified nucleotides and related methods
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
HUE039189T2 (hu) 2013-03-15 2018-12-28 Translate Bio Inc Nukleinsavak bejuttatásának szinergista fokozása kevert kiszerelésekkel
CN103214541A (zh) * 2013-04-19 2013-07-24 中国科学院上海有机化学研究所 含有天然胆固醇和赖氨酸类脂质阳离子有机功能分子及其脂质体、制备方法和应用
US9315472B2 (en) 2013-05-01 2016-04-19 Massachusetts Institute Of Technology 1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
US20160194625A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
EP4276176A3 (en) * 2013-10-22 2024-01-10 Translate Bio, Inc. Mrna therapy for argininosuccinate synthetase deficiency
JP6525435B2 (ja) 2013-10-22 2019-06-12 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 メッセンジャーｒｎａの送達のための脂質製剤
CA2928040A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Cns delivery of mrna and uses thereof
EP3985118A1 (en) 2013-11-22 2022-04-20 MiNA Therapeutics Limited C/ebp alpha short activating rna compositions and methods of use
ES2774552T3 (es) 2014-03-24 2020-07-21 Translate Bio Inc Terapia de ARNm para el tratamiento de enfermedades oculares
SI3134131T1 (sl) 2014-04-23 2022-04-29 Modernatx, Inc. Cepiva na osnovi nukleinskih kislin
JP6571679B2 (ja) 2014-04-25 2019-09-04 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の精製方法
ES2750686T3 (es) 2014-05-30 2020-03-26 Translate Bio Inc Lípidos biodegradables para la administración de ácidos nucleicos
EA033966B1 (ru) 2014-06-24 2019-12-13 Транслейт Био, Инк. Стереохимически обогащенные композиции для доставки нуклеиновых кислот
JP6782171B2 (ja) 2014-07-02 2020-11-11 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 メッセンジャーｒｎａのカプセル化
WO2016004202A1 (en) 2014-07-02 2016-01-07 Massachusetts Institute Of Technology Polyamine-fatty acid derived lipidoids and uses thereof
JP2017524357A (ja) 2014-07-16 2017-08-31 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． キメラポリヌクレオチド
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
WO2016079197A1 (en) * 2014-11-18 2016-05-26 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Delivery enhancers for conjugated sirna and lipid nanoparticles
MA56412A (fr) 2014-12-05 2022-05-04 Translate Bio Inc Thérapie par l'arn messager pour le traitement des maladies articulaires
CA2979695A1 (en) 2015-03-19 2016-09-22 Translate Bio, Inc. Mrna therapy for pompe disease
US10653768B2 (en) 2015-04-13 2020-05-19 Curevac Real Estate Gmbh Method for producing RNA compositions
CN107567497A (zh) * 2015-04-17 2018-01-09 库瑞瓦格股份公司 Rna的冻干
CN107530448A (zh) 2015-05-20 2018-01-02 库瑞瓦格股份公司 包含长链rna的干粉组合物
SG11201708540VA (en) * 2015-05-20 2017-12-28 Curevac Ag Dry powder composition comprising long-chain rna
PT3310764T (pt) 2015-06-19 2023-07-11 Massachusetts Inst Technology 2,5-piperazinadionas substituídas por alquenilo e o seu uso em composições para entregar um agente a um sujeito ou célula
RS61607B1 (sr) * 2015-07-22 2021-04-29 Nitto Denko Corp Kompozicije i postupci za nanočestične liofilne oblike
EP3736261B1 (en) 2015-09-17 2023-10-11 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
AU2016336344A1 (en) 2015-10-05 2018-04-19 Modernatx, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
JP6921833B2 (ja) 2015-10-22 2021-08-18 モデルナティーエックス， インコーポレイテッド ヒトサイトメガロウイルスワクチン
MD3386484T2 (ro) 2015-12-10 2022-11-30 Modernatx Inc Compoziții și metode de livrare a unor agenți terapeutici
ES2913626T3 (es) 2015-12-22 2022-06-03 Modernatx Inc Compuestos y composiciones para la administración intracelular de agentes
HRP20220525T1 (hr) 2015-12-23 2022-05-27 Modernatx, Inc. Postupci uporabe polinukleotida koji kodiraju ligand ox40
WO2017120612A1 (en) 2016-01-10 2017-07-13 Modernatx, Inc. Therapeutic mrnas encoding anti ctla-4 antibodies
KR102369898B1 (ko) 2016-04-08 2022-03-03 트랜슬레이트 바이오 인코포레이티드 다량체 코딩 핵산 및 그 용도
WO2017201332A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding acyl-coa dehydrogenase, very long-chain for the treatment of very long-chain acyl-coa dehydrogenase deficiency
EP3458590B9 (en) 2016-05-18 2021-08-18 ModernaTX, Inc. Polynucleotides encoding -galactosidase a for the treatment of fabry disease
US11801227B2 (en) 2016-05-18 2023-10-31 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
EP3458034A4 (en) 2016-05-18 2020-01-01 ModernaTX, Inc. POLYNUCLEOTIDS FOR THE CODING OF RELAXIN
WO2017201342A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
CN107441506A (zh) * 2016-05-30 2017-12-08 上海交通大学 基因输送载体及其制备与应用
US10835583B2 (en) 2016-06-13 2020-11-17 Translate Bio, Inc. Messenger RNA therapy for the treatment of ornithine transcarbamylase deficiency
AU2017347837A1 (en) 2016-10-26 2019-06-06 Modernatx, Inc. Messenger ribonucleic acids for enhancing immune responses and methods of use thereof
US11583504B2 (en) 2016-11-08 2023-02-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
JP2019533707A (ja) 2016-11-10 2019-11-21 トランスレイト バイオ， インコーポレイテッド Ｍｒｎａ担持脂質ナノ粒子を調製する改善されたプロセス
EP4249501A3 (en) 2017-01-09 2024-01-03 Whitehead Institute for Biomedical Research Methods of altering gene expression by perturbing transcription factor multimers that structure regulatory loops
CN110430894A (zh) 2017-02-01 2019-11-08 莫得纳特斯公司 编码活化致癌基因突变肽的免疫调节治疗性mrna组合物
HUE059025T2 (hu) 2017-02-27 2022-10-28 Translate Bio Inc Módszerek a hírvívõ RNS tisztítására
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
ES2925083T3 (es) 2017-02-27 2022-10-13 Translate Bio Inc Métodos de purificación de ARN mensajero
EP3592728A1 (en) 2017-03-07 2020-01-15 Translate Bio, Inc. Polyanionic delivery of nucleic acids
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
PL3596041T3 (pl) 2017-03-15 2023-03-06 Modernatx, Inc. Związek i kompozycje do dokomórkowego dostarczania środków terapeutycznych
ES2911186T3 (es) 2017-03-15 2022-05-18 Modernatx Inc Formas cristalinas de aminolípidos
AU2018268859A1 (en) 2017-05-16 2019-12-12 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding CFTR
US11485972B2 (en) 2017-05-18 2022-11-01 Modernatx, Inc. Modified messenger RNA comprising functional RNA elements
MA49395A (fr) 2017-06-14 2020-04-22 Modernatx Inc Polynucléotides codant pour le facteur viii de coagulation
WO2018232357A1 (en) 2017-06-15 2018-12-20 Modernatx, Inc. Rna formulations
WO2018236849A1 (en) 2017-06-19 2018-12-27 Translate Bio, Inc. MESSENGER RNA THERAPY FOR THE TREATMENT OF FRIEDREICH ATAXIA
MA50751A (fr) 2017-08-18 2020-06-24 Modernatx Inc Vaccins à base d'arnm efficaces
WO2019046809A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. METHODS OF MANUFACTURING LIPID NANOPARTICLES
US11447773B2 (en) 2017-09-08 2022-09-20 Mina Therapeutics Limited Stabilized HNF4A saRNA compositions and methods of use
WO2019048645A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
WO2019094702A1 (en) 2017-11-10 2019-05-16 Cocoon Biotech Inc. Ocular applications of silk-based products
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
JP7424976B2 (ja) 2017-11-22 2024-01-30 モダーナティエックス・インコーポレイテッド プロピオン酸血症の治療用のプロピオニルＣｏＡカルボキシラーゼアルファ及びベータサブユニットをコードするポリヌクレオチド
US11859215B2 (en) 2017-11-22 2024-01-02 Modernatx, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
US11167043B2 (en) 2017-12-20 2021-11-09 Translate Bio, Inc. Composition and methods for treatment of ornithine transcarbamylase deficiency
WO2019136241A1 (en) 2018-01-05 2019-07-11 Modernatx, Inc. Polynucleotides encoding anti-chikungunya virus antibodies
US11684577B2 (en) 2018-01-18 2023-06-27 Etherna Immunotherapies Nv Lipid nanoparticles
EP3746496A1 (en) 2018-02-02 2020-12-09 Translate Bio, Inc. Cationic polymers
EP3773745A1 (en) 2018-04-11 2021-02-17 ModernaTX, Inc. Messenger rna comprising functional rna elements
US11566246B2 (en) 2018-04-12 2023-01-31 Mina Therapeutics Limited SIRT1-saRNA compositions and methods of use
JP7448488B2 (ja) * 2018-05-15 2024-03-12 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
CN112384523A (zh) 2018-05-16 2021-02-19 川斯勒佰尔公司 核糖阳离子脂质
WO2019226650A1 (en) 2018-05-23 2019-11-28 Modernatx, Inc. Delivery of dna
CN112437767B (zh) 2018-05-24 2023-10-27 川斯勒佰尔公司 硫酯阳离子脂质
JP7441802B2 (ja) 2018-05-30 2024-03-01 トランスレイト バイオ， インコーポレイテッド ビタミンカチオン性脂質
EP3801627A1 (en) 2018-05-30 2021-04-14 Translate Bio, Inc. Phosphoester cationic lipids
CA3101454A1 (en) 2018-05-30 2019-12-05 Translate Bio, Inc. Messenger rna vaccines and uses thereof
WO2019232208A1 (en) 2018-05-30 2019-12-05 Translate Bio, Inc. Cationic lipids comprising a steroidal moiety
EP3826608A1 (en) 2018-07-23 2021-06-02 Translate Bio, Inc. Dry power formulations for messenger rna
WO2020023390A1 (en) 2018-07-25 2020-01-30 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
US11174500B2 (en) 2018-08-24 2021-11-16 Translate Bio, Inc. Methods for purification of messenger RNA
CN112839639A (zh) 2018-08-29 2021-05-25 川斯勒佰尔公司 制备负载mRNA的脂质纳米颗粒的改进方法
EP3846776A1 (en) 2018-09-02 2021-07-14 ModernaTX, Inc. Polynucleotides encoding very long-chain acyl-coa dehydrogenase for the treatment of very long-chain acyl-coa dehydrogenase deficiency
JP2022500436A (ja) 2018-09-13 2022-01-04 モダーナティエックス・インコーポレイテッドModernaTX, Inc. 糖原病を処置するためのグルコース−６−ホスファターゼをコードするポリヌクレオチド
MA53608A (fr) 2018-09-13 2021-07-21 Modernatx Inc Polynucléotides codant pour les sous-unités e1-alpha, e1-beta et e2 du complexe alpha-cétoacide déshydrogénase à chaîne ramifiée pour le traitement de la leucinose
WO2020056239A1 (en) 2018-09-14 2020-03-19 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
US20230145188A1 (en) 2018-09-14 2023-05-11 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia
WO2020061284A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Peg lipids and uses thereof
WO2020061295A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. High-purity peg lipids and uses thereof
MA53734A (fr) 2018-09-27 2021-08-04 Modernatx Inc Polynucléotides codant pour l'arginase 1 pour le traitement d'une déficience en arginase
CN113166783A (zh) 2018-10-09 2021-07-23 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
AU2019362031A1 (en) * 2018-10-19 2021-04-08 Translate Bio, Inc. Pumpless encapsulation of messenger RNA
WO2020097509A1 (en) 2018-11-08 2020-05-14 Translate Bio, Inc. Methods and compositions for messenger rna purification
US20220001026A1 (en) 2018-11-08 2022-01-06 Modernatx, Inc. Use of mrna encoding ox40l to treat cancer in human patients
EP3877368A1 (en) 2018-11-09 2021-09-15 Translate Bio, Inc. 2,5-dioxopiperazine lipids with intercalated ester, thioester, disulfide and anhydride moieities
WO2020097511A2 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. Messenger rna therapy for treatment of ocular diseases
CA3117866A1 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. Multi-peg lipid compounds
WO2020097379A2 (en) 2018-11-09 2020-05-14 Translate Bio, Inc. Peg lipidoid compounds
CA3119449A1 (en) 2018-11-12 2020-05-22 Translate Bio, Inc. Methods for inducing immune tolerance
WO2020106903A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. Cationic lipid compounds and compositions thereof for use in the delivery of messenger rna
CA3120647A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr
SG11202105367QA (en) * 2018-12-05 2021-06-29 Intellia Therapeutics Inc Modified amine lipids
AU2020206109A1 (en) 2019-01-07 2021-07-29 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
EP3953473A1 (en) 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
US20220177423A1 (en) 2019-04-18 2022-06-09 Translate Bio, Inc. Cystine cationic lipids
US20220233444A1 (en) 2019-04-22 2022-07-28 Translate Bio, Inc. Thioester cationic lipids
WO2020227085A1 (en) 2019-05-03 2020-11-12 Translate Bio, Inc. Di-thioester cationic lipids
CN114375190A (zh) 2019-05-08 2022-04-19 阿斯利康(瑞典)有限公司 用于皮肤和伤口的组合物及其使用方法
MA55899A (fr) 2019-05-08 2022-03-16 Modernatx Inc Polynucléotides codant pour la méthylmalonyl-coa mutase pour le traitement de l'acidémie méthylmalonique
SG11202112574RA (en) 2019-05-14 2021-12-30 Translate Bio Inc Improved process of preparing mrna-loaded lipid nanoparticles
IL310266A (en) 2019-05-22 2024-03-01 Massachusetts Inst Technology Circular Rana preparations and methods
CA3142608A1 (en) 2019-06-04 2020-12-10 Cocoon Biotech Inc. Silk-based products, formulations, and methods of use
WO2020257611A1 (en) 2019-06-21 2020-12-24 Translate Bio, Inc. Cationic lipids comprising an hydroxy moiety
JP2022537580A (ja) 2019-06-21 2022-08-26 トランスレイト バイオ， インコーポレイテッド トリシンおよびクエン酸脂質
MA56539A (fr) 2019-06-24 2022-04-27 Modernatx Inc Arn messager résistant à l'endonucléase et utilisations correspondantes
US20220387628A1 (en) 2019-06-24 2022-12-08 Modernatx, Inc. Messenger rna comprising functional rna elements and uses thereof
AU2020311364A1 (en) 2019-07-08 2022-02-03 Translate Bio, Inc. Improved mRNA-loaded lipid nanoparticles and processes of making the same
JP2022542562A (ja) 2019-07-23 2022-10-05 トランスレイト バイオ， インコーポレイテッド ｍＲＮＡ担持脂質ナノ粒子の安定性組成物および作製のプロセス
AU2020322014A1 (en) 2019-07-30 2022-02-24 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
WO2021026310A1 (en) 2019-08-06 2021-02-11 L.E.A.F. Holdings Group Llc Processes of preparing polyglutamated antifolates and uses of their compositions
CN114728887A (zh) 2019-09-19 2022-07-08 摩登纳特斯有限公司 用于治疗剂的细胞内递送的支链尾端脂质化合物和组合物
CA3155003A1 (en) 2019-09-20 2021-03-25 Translate Bio, Inc. Mrna encoding engineered cftr
AU2020355000A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating apolipoprotein B (APOB) gene expression
CN114729376A (zh) 2019-09-23 2022-07-08 欧米茄治疗公司 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法
JPWO2021060440A1 (da) * 2019-09-26 2021-04-01
WO2021072172A1 (en) 2019-10-09 2021-04-15 Translate Bio, Inc. Compositions, methods and uses of messenger rna
WO2021081058A1 (en) 2019-10-21 2021-04-29 Translate Bio, Inc. Compositions, methods and uses of messenger rna
EP4289951A3 (en) 2019-12-04 2024-03-13 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2021113806A1 (en) 2019-12-05 2021-06-10 Genzyme Corporation Arylamides and methods of use thereof
WO2021113809A1 (en) 2019-12-05 2021-06-10 Genzyme Corporation Arylamides and methods of use thereof
BR112022012085A2 (pt) 2019-12-20 2022-08-30 Translate Bio Inc Adminstração retal de rna mensageiro
JP2023508881A (ja) 2019-12-20 2023-03-06 トランスレイト バイオ， インコーポレイテッド Ｍｒｎａ担持脂質ナノ粒子を調製する改善されたプロセス
WO2021142245A1 (en) 2020-01-10 2021-07-15 Translate Bio, Inc. Compounds, pharmaceutical compositions and methods for modulating expression of muc5b in lung cells and tissues
IL294624A (en) * 2020-01-21 2022-09-01 Etherna Immunotherapies Nv lipid nanoparticles
US11576966B2 (en) 2020-02-04 2023-02-14 CureVac SE Coronavirus vaccine
JP2023514450A (ja) 2020-02-25 2023-04-05 トランスレイト バイオ， インコーポレイテッド Ｍｒｎａ担持脂質ナノ粒子を調製する改善された方法
CA3173745A1 (en) * 2020-02-27 2021-09-02 The Trustees Of Indiana University Cell-targeted nanoparticles to inhibit rna cargo
JP2023517326A (ja) 2020-03-11 2023-04-25 オメガ セラピューティクス， インコーポレイテッド フォークヘッドボックスｐ３（ｆｏｘｐ３）遺伝子発現をモジュレートするための組成物および方法
WO2021226463A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
EP4146265A1 (en) 2020-05-07 2023-03-15 Translate Bio, Inc. Optimized nucleotide sequences encoding sars-cov-2 antigens
WO2021226468A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Improved compositions for cftr mrna therapy
WO2021231697A1 (en) 2020-05-14 2021-11-18 Translate Bio, Inc. Peg lipidoid compounds
AU2021273502A1 (en) 2020-05-15 2023-02-02 Translate Bio, Inc. Lipid nanoparticle formulations for mRNA delivery
JP2023527309A (ja) 2020-05-19 2023-06-28 オルナ セラピューティクス インコーポレイテッド 環状ｒｎａ組成物及び方法
US20230235298A1 (en) 2020-06-01 2023-07-27 Modernatx, Inc. Phenylalanine hydroxylase variants and uses thereof
BR112022025217A2 (pt) * 2020-06-11 2023-01-03 Etherna Immunotherapies Nv Nanopartículas lipídicas
EP4165183A1 (en) 2020-06-12 2023-04-19 University of Rochester Encoding and expression of ace-trnas
WO2022006527A1 (en) 2020-07-02 2022-01-06 Maritime Therapeutics, Inc. Compositions and methods for reverse gene therapy
TW202214215A (zh) 2020-08-06 2022-04-16 美商現代公司 用於將有效負載分子遞送至氣道上皮之組成物
EP4225268A1 (en) 2020-10-06 2023-08-16 Translate Bio, Inc. Improved process and formulation of lipid nanoparticles
WO2022081548A1 (en) 2020-10-12 2022-04-21 Translate Bio, Inc. Improved process of preparing ice-based lipid nanoparticles
EP4225272A1 (en) 2020-10-12 2023-08-16 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
US20230365618A1 (en) * 2020-10-12 2023-11-16 Durect Corporation Crystalline thiocholesterol
US11771652B2 (en) 2020-11-06 2023-10-03 Sanofi Lipid nanoparticles for delivering mRNA vaccines
EP4240326A1 (en) 2020-11-09 2023-09-13 Translate Bio, Inc. Improved compositions for delivery of codon-optimized mrna
IL302625A (en) 2020-11-13 2023-07-01 Modernatx Inc Cystic fibrosis-encoding polynucleotides transmembrane conductance regulator for the treatment of cystic fibrosis
US20220287966A1 (en) 2020-11-25 2022-09-15 Translate Bio, Inc. Stable Liquid Lipid Nanoparticle Formulations
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
MX2023007574A (es) 2020-12-22 2023-09-29 CureVac SE "vacuna de arn contra variantes de sars-cov-2.
EP4277929A1 (en) 2021-01-14 2023-11-22 Translate Bio, Inc. Methods and compositions for delivering mrna coded antibodies
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
WO2022204380A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits and uses thereof
WO2022204390A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding phenylalanine hydroxylase and uses thereof
WO2022204369A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
WO2022204371A1 (en) 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles containing polynucleotides encoding glucose-6-phosphatase and uses thereof
JP2024512026A (ja) 2021-03-24 2024-03-18 モデルナティエックス インコーポレイテッド オルニチントランスカルバミラーゼ欠損症の治療を目的とした脂質ナノ粒子及びオルニチントランスカルバミラーゼをコードするポリヌクレオチド
EP4313115A1 (en) 2021-03-25 2024-02-07 Translate Bio, Inc. Optimized nucleotide sequences encoding the extracellular domain of human ace2 protein or a portion thereof
WO2022200810A1 (en) 2021-03-26 2022-09-29 Mina Therapeutics Limited Tmem173 sarna compositions and methods of use
JP2024515668A (ja) 2021-04-19 2024-04-10 トランスレイト バイオ， インコーポレイテッド Ｍｒｎａの送達のための改善された組成物
EP4329731A1 (en) * 2021-04-29 2024-03-06 ModernaTX, Inc. Lyophilization methods for preparing lipid formulated therapeutics
WO2022240806A1 (en) 2021-05-11 2022-11-17 Modernatx, Inc. Non-viral delivery of dna for prolonged polypeptide expression in vivo
WO2022246020A1 (en) 2021-05-19 2022-11-24 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia
WO2022266083A2 (en) 2021-06-15 2022-12-22 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
WO2022271776A1 (en) 2021-06-22 2022-12-29 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
JP2023003757A (ja) 2021-06-24 2023-01-17 株式会社アシックス 緩衝材、靴底および靴
CN117881395A (zh) 2021-07-01 2024-04-12 翻译生物公司 用于递送mRNA的组合物
EP4367242A2 (en) 2021-07-07 2024-05-15 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
WO2023287751A1 (en) 2021-07-12 2023-01-19 Modernatx, Inc. Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia
WO2023009499A1 (en) 2021-07-27 2023-02-02 Modernatx, Inc. Polynucleotides encoding glucose-6-phosphatase for the treatment of glycogen storage disease type 1a (gsd1a)
WO2023015261A1 (en) 2021-08-04 2023-02-09 Modernatx, Inc. Mrnas encoding chimeric metabolic reprogramming polypeptides and uses thereof
WO2023034946A1 (en) 2021-09-03 2023-03-09 Genzyme Corporation Indole compounds and uses thereof in the treatement of cystic fibrosis
IL311038A (en) 2021-09-03 2024-04-01 Genzyme Corp Indole compounds and methods of use
IL309505A (en) 2021-09-03 2024-02-01 CureVac SE Lipid nanoparticles for nucleic acid delivery
CA3132191A1 (en) * 2021-09-28 2023-03-28 Providence Therapeutics Holdings Inc. Compositions and methods for the prevention and/or treatment of covid-19
WO2023056044A1 (en) 2021-10-01 2023-04-06 Modernatx, Inc. Polynucleotides encoding relaxin for the treatment of fibrosis and/or cardiovascular disease
WO2023073228A1 (en) 2021-10-29 2023-05-04 CureVac SE Improved circular rna for expressing therapeutic proteins
WO2023077170A1 (en) 2021-11-01 2023-05-04 Modernatx, Inc. Polynucleotides encoding integrin beta-6 and methods of use thereof
CA3236235A1 (en) 2021-11-08 2023-05-11 Orna Therapeutics, Inc. Lipid nanoparticle compositions for delivering circular polynucleotides
WO2023086893A1 (en) 2021-11-10 2023-05-19 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
WO2023086465A1 (en) 2021-11-12 2023-05-19 Modernatx, Inc. Compositions for the delivery of payload molecules to airway epithelium
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
WO2023141586A1 (en) 2022-01-21 2023-07-27 Orna Therapeutics, Inc. Systemic administration of circular rna polynucleotides encoding muscle proteins or protein complexes
WO2023144330A1 (en) 2022-01-28 2023-08-03 CureVac SE Nucleic acid encoded transcription factor inhibitors
WO2023150753A1 (en) 2022-02-07 2023-08-10 University Of Rochester Optimized sequences for enhanced trna expression or/and nonsense mutation suppression
WO2023154818A1 (en) 2022-02-09 2023-08-17 Modernatx, Inc. Mucosal administration methods and formulations
TW202345864A (zh) 2022-02-18 2023-12-01 美商現代公司 編碼檢查點癌症疫苗之ｍＲＮＡ及其用途
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
WO2023177904A1 (en) 2022-03-18 2023-09-21 Modernatx, Inc. Sterile filtration of lipid nanoparticles and filtration analysis thereof for biological applications
WO2023183909A2 (en) 2022-03-25 2023-09-28 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
WO2023196399A1 (en) 2022-04-06 2023-10-12 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding argininosuccinate lyase for the treatment of argininosuccinic aciduria
WO2023215498A2 (en) 2022-05-05 2023-11-09 Modernatx, Inc. Compositions and methods for cd28 antagonism
WO2023227608A1 (en) 2022-05-25 2023-11-30 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
WO2023231959A2 (en) 2022-05-30 2023-12-07 Shanghai Circode Biomed Co., Ltd Synthetic circular rna compositions and methods of use thereof
WO2024008967A1 (en) * 2022-07-08 2024-01-11 Polyplus Transfection Lipid nanoparticles comprising an imidazolium-based cationic lipid
WO2024026254A1 (en) 2022-07-26 2024-02-01 Modernatx, Inc. Engineered polynucleotides for temporal control of expression
WO2024044147A1 (en) 2022-08-23 2024-02-29 Modernatx, Inc. Methods for purification of ionizable lipids
WO2024054851A1 (en) 2022-09-07 2024-03-14 Sionna Therapeutics Macrocyclic compounds, compositions and methods of using thereof
WO2024054845A1 (en) 2022-09-07 2024-03-14 Sionna Therapeutics Macrocycic compounds, compositions, and methods of using thereof
WO2024054840A1 (en) 2022-09-07 2024-03-14 Sionna Therapeutics Macrocyclic compounds, compositions, and methods of using thereof
US20240156949A1 (en) 2022-10-28 2024-05-16 Glaxosmithkline Biologicals Sa Nucleic Acid Based Vaccine
WO2024097639A1 (en) 2022-10-31 2024-05-10 Modernatx, Inc. Hsa-binding antibodies and binding proteins and uses thereof
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024102677A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Circular rna compositions
WO2024102730A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and nanoparticle compositions for delivering polynucleotides
WO2024112652A1 (en) 2022-11-21 2024-05-30 Translate Bio, Inc. Compositions of dry powder formulations of messenger rna and methods of use thereof
WO2024118866A1 (en) 2022-12-01 2024-06-06 Modernatx, Inc. Gpc3-specific antibodies, binding domains, and related proteins and uses thereof
US11833224B1 (en) 2023-02-08 2023-12-05 Leuvian Llc Lyoprotectant compositions and uses thereof

Family Cites Families (128)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5223263A (en) 1988-07-07 1993-06-29 Vical, Inc. Liponucleotide-containing liposomes
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
DE3728917A1 (de) * 1987-08-29 1989-03-09 Roth Hermann J Neue lipide mit unsymmetrisch substituierter disulfidbruecke
US4946683A (en) 1987-11-18 1990-08-07 Vestar, Inc. Multiple step entrapment/loading procedure for preparing lipophilic drug-containing liposomes
US5948441A (en) 1988-03-07 1999-09-07 The Liposome Company, Inc. Method for size separation of particles
US6214804B1 (en) * 1989-03-21 2001-04-10 Vical Incorporated Induction of a protective immune response in a mammal by injecting a DNA sequence
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5194654A (en) 1989-11-22 1993-03-16 Vical, Inc. Lipid derivatives of phosphonoacids for liposomal incorporation and method of use
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
CA2168656C (en) * 1993-08-06 2007-09-18 Yechezkel Barenholz A method for preparation of vesicles loaded with biological structures, biopolymers and/or oligomers
EP0713387B1 (en) 1993-08-06 1998-03-04 Opperbas Holding B.V. A method for preparation of vesicles loaded with biological structures, biopolymers and/or oligomers
JPH08511956A (ja) 1994-04-07 1996-12-17 アクゾ・ノベル・エヌ・ベー Ｒｎａを含む凍結乾燥組成物
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5965434A (en) * 1994-12-29 1999-10-12 Wolff; Jon A. Amphipathic PH sensitive compounds and delivery systems for delivering biologically active compounds
US5744335A (en) 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
US5677124A (en) 1996-07-03 1997-10-14 Ambion, Inc. Ribonuclease resistant viral RNA standards
CA2289702C (en) 1997-05-14 2008-02-19 Inex Pharmaceuticals Corp. High efficiency encapsulation of charged therapeutic agents in lipid vesicles
EP1021549A2 (en) 1997-09-19 2000-07-26 Sequitur, Inc. SENSE mRNA THERAPY
CN100471522C (zh) 1998-03-13 2009-03-25 惠氏 聚核苷酸组合物及其制备方法与用途
US6169078B1 (en) 1998-05-12 2001-01-02 University Of Florida Materials and methods for the intracellular delivery of substances
US6379698B1 (en) 1999-04-06 2002-04-30 Isis Pharmaceuticals, Inc. Fusogenic lipids and vesicles
EP1880736A1 (en) 1999-04-23 2008-01-23 Alza Corporation Releasable linkage and composition containing same
AU6105300A (en) 1999-07-16 2001-02-05 Purdue Research Foundation Vinyl ether lipids with cleavable hydrophilic headgroups
DE50211110D1 (de) 2001-04-21 2007-12-06 Curevac Gmbh INJEKTIONSGERÄT FÜR mRNA APPLIKATION
US7514099B2 (en) 2005-02-14 2009-04-07 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
WO2004033620A2 (en) 2001-11-02 2004-04-22 Insert Therapeutics, Inc. Methods and compositions for therapeutic use of rna interference
US7994149B2 (en) * 2003-02-03 2011-08-09 Medtronic, Inc. Method for treatment of Huntington's disease through intracranial delivery of sirna
EP1613285A2 (en) 2003-03-31 2006-01-11 Alza Corporation Lipid particles having asymmetric lipid coating and method of preparing same
AU2004272646B2 (en) 2003-09-15 2011-11-24 Arbutus Biopharma Corporation Polyethyleneglycol-modified lipid compounds and uses thereof
US7799565B2 (en) 2004-06-07 2010-09-21 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
WO2005120152A2 (en) 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
EP1811960A2 (en) * 2004-11-19 2007-08-01 Novosom AG Improvements in or relating to pharmaceutical compositions for local administration
EP1743654A1 (en) 2005-07-15 2007-01-17 TopoTarget Germany AG Use of inhibitors of histone deacetylases in combination with NSAID for the therapy of cancer and/or inflammatory diseases
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
JP5336853B2 (ja) 2005-11-02 2013-11-06 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 修飾ｓｉＲＮＡ分子およびその使用法
FR2904144A1 (fr) 2006-07-19 2008-01-25 St Microelectronics Rousset Procede de fabrication d'un wafer de semi-conducteur comprenant un filtre optique integre
US7776840B2 (en) 2007-02-21 2010-08-17 Cutanea Life Sciences, Inc. Methods of use of biomaterial and injectable implant containing biomaterial
WO2009026328A2 (en) 2007-08-21 2009-02-26 Immune Disease Institute, Inc. Methods of delivery of agents to leukocytes and endothelial cells
WO2009030254A1 (en) 2007-09-04 2009-03-12 Curevac Gmbh Complexes of rna and cationic peptides for transfection and for immunostimulation
WO2009120247A2 (en) * 2007-12-27 2009-10-01 The Ohio State University Research Foundation Lipid nanoparticle compositions and methods of making and using the same
JP2011520962A (ja) * 2008-05-19 2011-07-21 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル 新規カチオン性脂質を含む方法及び組成物
WO2010019718A2 (en) 2008-08-13 2010-02-18 California Institute Of Technology Carrier nanoparticles and related compositions, methods and systems
US9139554B2 (en) 2008-10-09 2015-09-22 Tekmira Pharmaceuticals Corporation Amino lipids and methods for the delivery of nucleic acids
WO2010053572A2 (en) 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
NO2355851T3 (da) 2008-11-10 2018-09-01
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
EP2823810B9 (en) * 2009-07-09 2019-10-30 Novosom Verwaltungs GmbH Amphoteric liposomes comprising imino lipids
US20120195936A1 (en) 2009-07-31 2012-08-02 Ethris Gmbh Rna with a combination of unmodified and modified nucleotides for protein expression
US8311261B2 (en) 2009-08-14 2012-11-13 Graber Curtis E Acoustic transducer array
DE102009043342A1 (de) * 2009-09-29 2011-03-31 Bayer Technology Services Gmbh Stoffe für selbstorganisierte Träger zur kontrollierten Freisetzung eines Wirkstoffs
PT2506857T (pt) 2009-12-01 2018-05-14 Translate Bio Inc Entrega de arnm para o acréscimo de proteínas e enzimas em doenças genéticas humanas
US9687550B2 (en) * 2009-12-07 2017-06-27 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
WO2011069529A1 (en) 2009-12-09 2011-06-16 Curevac Gmbh Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
WO2011069528A1 (en) 2009-12-09 2011-06-16 Curevac Gmbh Lyophilization of nucleic acids in lactate-containing solutions
EP2387999A1 (en) 2010-05-21 2011-11-23 CureVac GmbH Histidine-containing solution for transfection and/or injection of nucleic acids and uses thereof
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
US8710200B2 (en) 2011-03-31 2014-04-29 Moderna Therapeutics, Inc. Engineered nucleic acids encoding a modified erythropoietin and their expression
AU2012267578B2 (en) * 2011-06-08 2017-04-20 Translate Bio, Inc. Cleavable lipids
CA3107288A1 (en) 2011-06-08 2012-12-13 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mrna delivery
US9387236B2 (en) 2011-06-10 2016-07-12 Prothera Inc. Pharmaceutical compositions containing protease and methods for the treatment of lysosomal storage diseases
US8999950B2 (en) 2011-10-05 2015-04-07 Protiva Biotherapeutics Inc. Compositions and methods for silencing aldehyde dehydrogenase
CN107522664B (zh) 2011-10-27 2021-03-16 麻省理工学院 能够形成药物包封微球的在n末端上官能化的氨基酸衍生物
US20140343129A1 (en) 2011-12-14 2014-11-20 Moderna Therapeutics, Inc. Modified nucleic acids, and acute care uses thereof
EP2793906A4 (en) 2011-12-21 2016-01-13 Moderna Therapeutics Inc METHOD FOR INCREASING THE LIFE-LIFE OR DURABILITY OF AN ORGAN OR ORGAN EXPLOITATE
KR20220045089A (ko) 2012-02-24 2022-04-12 아뷰터스 바이오파마 코포레이션 트리알킬 양이온성 지질 및 그의 사용 방법
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
CA2868030C (en) 2012-03-29 2021-05-25 Shire Human Genetic Therapies, Inc. Lipid-derived neutral nanoparticles
CA2868034C (en) 2012-03-29 2021-07-27 Shire Human Genetic Therapies, Inc. Ionizable cationic lipids
WO2013151667A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides
US9303079B2 (en) 2012-04-02 2016-04-05 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US20150050354A1 (en) 2012-04-02 2015-02-19 Moderna Therapeutics, Inc. Modified polynucleotides for the treatment of otic diseases and conditions
EP3498267A1 (en) 2012-05-25 2019-06-19 CureVac AG Reversible immobilization and/or controlled release of nucleic acid containing nanoparticles by (biodegradable) polymer coatings
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
WO2014089486A1 (en) 2012-12-07 2014-06-12 Shire Human Genetic Therapies, Inc. Lipidic nanoparticles for mrna delivering
EP3750903A1 (en) 2013-03-14 2020-12-16 Translate Bio, Inc. Ribonucleic acids with 4'-thio-modified nucleotides and related methods
WO2014152940A1 (en) 2013-03-14 2014-09-25 Shire Human Genetic Therapies, Inc. Mrna therapeutic compositions and use to treat diseases and disorders
MX2015011945A (es) 2013-03-14 2015-12-01 Shire Human Genetic Therapies Evaluacion cuantitativa para la eficacia de los casquetes de arn mensajero.
HUE055044T2 (hu) 2013-03-14 2021-10-28 Translate Bio Inc MRNS-kódolt ellenanyagok bejuttatására szolgáló eljárások és készítmények
AU2014239250A1 (en) 2013-03-14 2015-08-27 Shire Human Genetic Therapies, Inc. Quantitative assessment for cap efficiency of messenger RNA
EA037922B1 (ru) 2013-03-14 2021-06-07 Шир Хьюман Дженетик Терапис, Инк. мРНК CFTR И КОМПОЗИЦИИ ДЛЯ ЛЕЧЕНИЯ МУКОВИСЦИДОЗА У МЛЕКОПИТАЮЩЕГО
EP2970955B1 (en) 2013-03-14 2018-11-14 Translate Bio, Inc. Methods for purification of messenger rna
HUE039189T2 (hu) 2013-03-15 2018-12-28 Translate Bio Inc Nukleinsavak bejuttatásának szinergista fokozása kevert kiszerelésekkel
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
EP3567112A1 (en) 2013-06-13 2019-11-13 Translate Bio, Inc. Messenger rna based viral production
ES2810298T3 (es) 2013-06-28 2021-03-08 Ethris Gmbh Composiciones para introducir ARN en células
SI3019619T1 (sl) 2013-07-11 2021-12-31 Modernatx, Inc. Sestave, ki zajemajo sintetične polinukleotide, ki kodirajo proteine, pozvezane s crispr, in sintetične sgrna, ter metode uporabe
EP3033325B1 (en) 2013-07-23 2019-12-04 Arbutus Biopharma Corporation Compositions and methods for delivering messenger rna
US20160201064A1 (en) 2013-08-16 2016-07-14 Rana Therapeutics, Inc. Compositions and methods for modulating expression of frataxin
EP3033422A4 (en) 2013-08-16 2017-08-02 Rana Therapeutics Inc. Oligonucleotides targeting euchromatin regions of genes
WO2015023975A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Compositions and methods for modulating rna
AU2014310932B2 (en) 2013-08-21 2019-06-06 CureVac SE Composition and vaccine for treating lung cancer
RU2016109938A (ru) 2013-08-21 2017-09-26 Куревак Аг Композиция и вакцина для лечения рака предстательной железы
EP3049065A1 (en) 2013-09-26 2016-08-03 BioNTech AG Particles comprising a shell with rna
JP6525435B2 (ja) 2013-10-22 2019-06-12 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 メッセンジャーｒｎａの送達のための脂質製剤
CA2928040A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Cns delivery of mrna and uses thereof
EP4276176A3 (en) 2013-10-22 2024-01-10 Translate Bio, Inc. Mrna therapy for argininosuccinate synthetase deficiency
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
EP3110954A1 (en) 2014-02-26 2017-01-04 Ethris GmbH Compositions for gastrointestinal administration of rna
ES2774552T3 (es) 2014-03-24 2020-07-21 Translate Bio Inc Terapia de ARNm para el tratamiento de enfermedades oculares
EP3129050A2 (en) 2014-04-01 2017-02-15 CureVac AG Polymeric carrier cargo complex for use as an immunostimulating agent or as an adjuvant
JP6571679B2 (ja) 2014-04-25 2019-09-04 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の精製方法
ES2750686T3 (es) 2014-05-30 2020-03-26 Translate Bio Inc Lípidos biodegradables para la administración de ácidos nucleicos
EA033966B1 (ru) 2014-06-24 2019-12-13 Транслейт Био, Инк. Стереохимически обогащенные композиции для доставки нуклеиновых кислот
JP6782171B2 (ja) 2014-07-02 2020-11-11 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 メッセンジャーｒｎａのカプセル化
MA56412A (fr) 2014-12-05 2022-05-04 Translate Bio Inc Thérapie par l'arn messager pour le traitement des maladies articulaires
CA2979695A1 (en) 2015-03-19 2016-09-22 Translate Bio, Inc. Mrna therapy for pompe disease
US10653768B2 (en) 2015-04-13 2020-05-19 Curevac Real Estate Gmbh Method for producing RNA compositions
CN107567497A (zh) 2015-04-17 2018-01-09 库瑞瓦格股份公司 Rna的冻干
DK3294885T3 (da) 2015-05-08 2020-08-10 Curevac Real Estate Gmbh Fremgangsmåde til at fremstille rna
WO2016184577A2 (en) 2015-05-20 2016-11-24 Curevac Ag Dry powder composition comprising long-chain rna
CN108473969B (zh) 2015-10-14 2022-09-13 川斯勒佰尔公司 用于增强生产的rna相关酶的修饰
KR102369898B1 (ko) 2016-04-08 2022-03-03 트랜슬레이트 바이오 인코포레이티드 다량체 코딩 핵산 및 그 용도
US10835583B2 (en) 2016-06-13 2020-11-17 Translate Bio, Inc. Messenger RNA therapy for the treatment of ornithine transcarbamylase deficiency
AU2017286606A1 (en) 2016-06-14 2018-12-13 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
JP2019533707A (ja) 2016-11-10 2019-11-21 トランスレイト バイオ， インコーポレイテッド Ｍｒｎａ担持脂質ナノ粒子を調製する改善されたプロセス
KR20190120160A (ko) 2016-11-10 2019-10-23 트랜슬레이트 바이오 인코포레이티드 Mrna의 전달을 위한 개선된 ice계 지질 나노입자 제형
ES2971902T3 (es) 2017-02-27 2024-06-10 Translate Bio Inc Síntesis a gran escala de ARN mensajero
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
HUE059025T2 (hu) 2017-02-27 2022-10-28 Translate Bio Inc Módszerek a hírvívõ RNS tisztítására
ES2925083T3 (es) 2017-02-27 2022-10-13 Translate Bio Inc Métodos de purificación de ARN mensajero
AU2018268859A1 (en) 2017-05-16 2019-12-12 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding CFTR
WO2018236849A1 (en) 2017-06-19 2018-12-27 Translate Bio, Inc. MESSENGER RNA THERAPY FOR THE TREATMENT OF FRIEDREICH ATAXIA
GB201717996D0 (en) 2017-10-31 2017-12-13 Portal Medical Ltd Medicament dispenser device
WO2019207060A1 (en) 2018-04-25 2019-10-31 Ethris Gmbh Lipid-based formulations for the delivery of rna
CA3120647A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr

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