CA2979695C - RNA THERAPY FOR POMPE DISEASE - Google Patents

RNA THERAPY FOR POMPE DISEASE

Info

Publication number
CA2979695C
CA2979695C CA2979695A CA2979695A CA2979695C CA 2979695 C CA2979695 C CA 2979695C CA 2979695 A CA2979695 A CA 2979695A CA 2979695 A CA2979695 A CA 2979695A CA 2979695 C CA2979695 C CA 2979695C
Authority
CA
Canada
Prior art keywords
mrna
composition
cholesterol
gaa
administering
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.)
Active
Application number
CA2979695A
Other languages
English (en)
French (fr)
Other versions
CA2979695A1 (en
Inventor
Frank DeRosa
Michael Heartlein
Original Assignee
Translate Bio Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Translate Bio Inc filed Critical Translate Bio Inc
Publication of CA2979695A1 publication Critical patent/CA2979695A1/en
Application granted granted Critical
Publication of CA2979695C publication Critical patent/CA2979695C/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P39/00—General protective or antinoxious agents
    • A61P39/02—Antidotes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Dispersion Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Diabetes (AREA)
  • Heart & Thoracic Surgery (AREA)
CA2979695A 2015-03-19 2016-03-17 RNA THERAPY FOR POMPE DISEASE Active CA2979695C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562135338P 2015-03-19 2015-03-19
US62/135,338 2015-03-19
PCT/US2016/022895 WO2016149508A1 (en) 2015-03-19 2016-03-17 Mrna therapy for pompe disease

Publications (2)

Publication Number Publication Date
CA2979695A1 CA2979695A1 (en) 2016-09-22
CA2979695C true CA2979695C (en) 2025-02-06

Family

ID=55755676

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2979695A Active CA2979695C (en) 2015-03-19 2016-03-17 RNA THERAPY FOR POMPE DISEASE

Country Status (6)

Country Link
US (4) US10172924B2 (https=)
EP (2) EP3270894B1 (https=)
JP (4) JP6895892B2 (https=)
AU (3) AU2016233135B2 (https=)
CA (1) CA2979695C (https=)
WO (1) WO2016149508A1 (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3354644A1 (en) 2011-06-08 2018-08-01 Translate Bio, Inc. Cleavable lipids
EP3585892B8 (en) 2017-02-27 2022-07-13 Translate Bio, Inc. Methods for purification of messenger rna
CA3053814A1 (en) 2017-02-27 2018-08-30 Translate Bio, Inc. Large scale synthesis of messenger rna
WO2018157133A1 (en) 2017-02-27 2018-08-30 Translate Bio, Inc. Methods for purification of messenger rna
US20220184185A1 (en) * 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
CA3108544A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
CN121253826A (zh) 2018-10-05 2026-01-02 西雅图儿童医院以西雅图儿童研究机构名义经营 针对原发性免疫缺陷、胱氨酸病和威尔逊病的新生儿筛查
CN113166783B (zh) 2018-10-09 2024-10-11 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
KR20210089648A (ko) 2018-11-08 2021-07-16 트랜슬레이트 바이오 인코포레이티드 메신저 rna 정제를 위한 방법 및 조성물
US10974851B2 (en) 2018-11-09 2021-04-13 Textron Innovations Inc. System and method for maintaining and configuring rotorcraft
JP2022516356A (ja) * 2019-01-07 2022-02-25 トランスレイト バイオ， インコーポレイテッド 原発性線毛機能不全症の治療のための組成物および方法
AU2021216055A1 (en) * 2020-02-08 2022-09-29 Genzyme Corporation Compositions and methods for treating pompe disease
EP4126966A4 (en) 2020-03-31 2024-08-07 Seattle Children's Hospital d/b/a Seattle Children's Research Institute PROTEOMIC SCREENING FOR LYSOSOMAL STORAGE DISEASES
CN115298215A (zh) 2020-04-02 2022-11-04 西雅图儿童医院以西雅图儿童研究机构名义经营 特异性地结合与原发性免疫缺陷：维斯科特-奥尔德里奇综合征和x连锁无丙种球蛋白血症相关的肽的抗体
JP2023546175A (ja) 2020-10-14 2023-11-01 ジョージ・メイソン・リサーチ・ファウンデーション・インコーポレイテッド 脂質ナノ粒子製造の方法及びそれに由来する組成物
EP4229191A1 (en) 2020-10-15 2023-08-23 Translate Bio, Inc. Large scale synthesis of messenger rna

Family Cites Families (117)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4401796A (en) 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
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
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US5153319A (en) 1986-03-31 1992-10-06 University Patents, Inc. Process for preparing polynucleotides
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5700642A (en) 1995-05-22 1997-12-23 Sri International Oligonucleotide sizing using immobilized cleavable primers
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
ATE285477T1 (de) 1995-06-07 2005-01-15 Inex Pharmaceutical Corp Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
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
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
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
AU2003223780A1 (en) * 2002-04-30 2003-11-17 University Of Florida Treatment for pompe disease
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
WO2004011647A1 (en) 2002-07-26 2004-02-05 Chiron Corporation Modified small interfering rna molecules and methods of use
ATE521701T1 (de) 2003-01-22 2011-09-15 Univ Duke Verbesserte konstrukte zur expression lysosomaler polypeptide
NZ592917A (en) 2003-09-15 2012-12-21 Protiva Biotherapeutics Inc Stable polyethyleneglycol (PEG) dialkyloxypropyl (DAA) lipid conjugates
WO2005077333A2 (en) * 2004-02-10 2005-08-25 University Of Florida Research Foundation, Inc. Gel-based delivery of recombinant adeno-associated virus vectors
EP2399924B1 (en) 2004-05-27 2015-01-28 Alnylam Pharmaceuticals, Inc. Nuclease resistant double-stranded ribonucleic acid
US7799565B2 (en) 2004-06-07 2010-09-21 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
US7314164B2 (en) 2004-07-01 2008-01-01 American Express Travel Related Services Company, Inc. System for biometric security using a smartcard
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
EP2049665A2 (en) 2006-07-28 2009-04-22 Applera Corporation Dinucleotide mrna cap analogs
US20120322861A1 (en) * 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
WO2009058911A2 (en) 2007-10-31 2009-05-07 Applied Biosystems Inc. Preparation and isolation of 5' capped mrna
WO2009082607A2 (en) 2007-12-04 2009-07-02 Alnylam Pharmaceuticals, Inc. Targeting lipids
WO2009082817A1 (en) 2007-12-27 2009-07-09 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering rna
SI3470077T1 (sl) * 2008-02-12 2020-12-31 Amicus Therapeutics, Inc. Postopek za napovedovanje odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
WO2009126933A2 (en) 2008-04-11 2009-10-15 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
CN104119242B (zh) 2008-10-09 2017-07-07 泰米拉制药公司 改善的氨基脂质和递送核酸的方法
ES2646630T3 (es) 2008-11-07 2017-12-14 Massachusetts Institute Of Technology Lipidoides aminoalcohólicos y usos de los mismos
HUE037082T2 (hu) 2008-11-10 2018-08-28 Arbutus Biopharma Corp Új lipidek és készítmények terápiás hatóanyagok szállítására
CA2750561C (en) 2009-01-26 2017-10-10 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
KR20180094137A (ko) 2009-05-05 2018-08-22 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
MX342785B (es) 2009-06-10 2016-10-12 Alnylam Pharmaceuticals Inc Formulacion mejorada de lipido.
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
US8236943B2 (en) 2009-07-01 2012-08-07 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011011447A1 (en) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression
HUE042177T2 (hu) 2009-12-01 2019-06-28 Translate Bio Inc Szteroidszármazék mRNS szállítására humán genetikai betegségekben
WO2011141705A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
RU2013120302A (ru) 2010-10-01 2014-11-20 Модерна Терапьютикс, Инк. Сконструированные нуклеиновые кислоты и способы их применения
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
EP2691101A2 (en) 2011-03-31 2014-02-05 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
WO2012158736A1 (en) 2011-05-17 2012-11-22 modeRNA Therapeutics Engineered nucleic acids and methods of use thereof for non-human vertebrates
PL3586861T3 (pl) 2011-06-08 2022-05-23 Translate Bio, Inc. Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna
EP3354644A1 (en) 2011-06-08 2018-08-01 Translate Bio, Inc. Cleavable lipids
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2755986A4 (en) 2011-09-12 2015-05-20 Moderna Therapeutics Inc MANIPULATED NUCLEIC ACIDS AND METHOD OF APPLICATION THEREFOR
EP3384938A1 (en) 2011-09-12 2018-10-10 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
US8999950B2 (en) 2011-10-05 2015-04-07 Protiva Biotherapeutics Inc. Compositions and methods for silencing aldehyde dehydrogenase
KR102451116B1 (ko) 2011-10-27 2022-10-06 메사추세츠 인스티튜트 오브 테크놀로지 약물 캡슐화 마이크로스피어를 형성할 수 있는, ｎ-말단 상에 관능화된 아미노산 유도체
US20140378538A1 (en) 2011-12-14 2014-12-25 Moderma Therapeutics, Inc. Methods of responding to a biothreat
WO2013090186A1 (en) 2011-12-14 2013-06-20 modeRNA Therapeutics Modified nucleic acids, and acute care uses thereof
CN104114572A (zh) 2011-12-16 2014-10-22 现代治疗公司 经修饰的核苷、核苷酸和核酸组合物
JP2015510495A (ja) 2011-12-21 2015-04-09 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 器官または器官移植片の生存可能性または寿命を延長する方法
EP2797634A4 (en) 2011-12-29 2015-08-05 Moderna Therapeutics Inc MODIFIED MRNAS FOR THE CODING OF CELL-PENETRATING POLYPEPTIDES
SG11201405157PA (en) 2012-02-24 2014-10-30 Protiva Biotherapeutics Inc Trialkyl cationic lipids and methods of use thereof
KR20200032244A (ko) * 2012-03-07 2020-03-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
US20150050354A1 (en) 2012-04-02 2015-02-19 Moderna Therapeutics, Inc. Modified polynucleotides for the treatment of otic diseases and conditions
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
US20140275229A1 (en) 2012-04-02 2014-09-18 Moderna Therapeutics, Inc. Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1
CA2868391A1 (en) 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US10648001B2 (en) * 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
NL2009659C2 (en) * 2012-10-18 2014-04-22 Avantium Technologies B V System and method for operating parallel reactors.
WO2014081507A1 (en) 2012-11-26 2014-05-30 Moderna Therapeutics, Inc. Terminally modified rna
EP4331620A3 (en) * 2012-12-07 2024-12-04 Translate Bio, Inc. Lipidic nanoparticles for mrna delivery
WO2014093574A1 (en) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified polynucleotides for altering cell phenotype
US20140200261A1 (en) 2013-01-17 2014-07-17 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
EP2981551B1 (en) * 2013-02-20 2020-06-03 Valerion Therapeutics, LLC Methods and compositions for treatment of pompe disease
US20160022774A1 (en) 2013-03-12 2016-01-28 Moderna Therapeutics, Inc. Diagnosis and treatment of fibrosis
EP2968391A1 (en) 2013-03-13 2016-01-20 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
KR20210122917A (ko) * 2013-03-14 2021-10-12 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
DK2970456T3 (da) * 2013-03-14 2021-07-05 Translate Bio Inc Fremgangsmåder og sammensætninger til levering af mrna-kodede antistoffer
WO2014152027A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Manufacturing methods for production of rna transcripts
WO2014152030A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Removal of dna fragments in mrna production process
US20160032273A1 (en) 2013-03-15 2016-02-04 Moderna Therapeutics, Inc. Characterization of mrna molecules
WO2014144711A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Analysis of mrna heterogeneity and stability
WO2014152031A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Ribonucleic acid purification
WO2014144767A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Ion exchange purification of mrna
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
SMT202100691T1 (it) 2013-07-11 2022-01-10 Modernatx Inc Composizioni 5 comprendenti polinucleotidi sintetici che codificano proteine correlate a crispr e sgrna sintetici e metodi d'uso
US20160346363A1 (en) * 2013-07-15 2016-12-01 New York University Methods and oral formulations for enzyme replacement therapy of human lysosomal and metabolic diseases
WO2015011633A1 (en) 2013-07-23 2015-01-29 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
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
WO2015036451A1 (en) * 2013-09-11 2015-03-19 Synthena Ag Nucleic acids and methods for the treatment of pompe disease
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052479A4 (en) 2013-10-02 2017-10-25 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
WO2015051169A2 (en) 2013-10-02 2015-04-09 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
AU2014337156A1 (en) 2013-10-18 2016-05-12 Modernatx, Inc. Compositions and methods for tolerizing cellular systems
EA034103B1 (ru) * 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
EP4039807A1 (en) * 2014-06-10 2022-08-10 Erasmus University Rotterdam Medical Center Antisense oligonucleotides useful in treatment of pompe disease
BR112017006679A2 (pt) 2014-10-02 2017-12-26 Protiva Biotherapeutics Inc moléculas, composição, partícula, composição farmacêutica, métodos para silenciar a expressão de um gene, usos de uma partícula, métodos para melhorar um ou mais sintomas, métodos para tratar uma infecção, usos de uma composição, método para inibir a replicação do vírus da hepatite d
WO2016071857A1 (en) 2014-11-07 2016-05-12 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus expression
EP3461904A1 (en) 2014-11-10 2019-04-03 ModernaTX, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
WO2016077123A1 (en) 2014-11-10 2016-05-19 Moderna Therapeutics, Inc. Multiparametric nucleic acid optimization
EP3233132A4 (en) 2014-12-19 2018-06-27 Modernatx, Inc. Terminal modifications of polynucleotides
KR102510941B1 (ko) * 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제

Also Published As

Publication number Publication date
AU2016233135B2 (en) 2021-07-08
US11712463B2 (en) 2023-08-01
AU2021232818A1 (en) 2021-10-14
JP7758708B2 (ja) 2025-10-22
CA2979695A1 (en) 2016-09-22
US20210401951A1 (en) 2021-12-30
AU2024202731A1 (en) 2024-05-16
US20160324940A1 (en) 2016-11-10
JP2026031545A (ja) 2026-02-24
US20190134164A1 (en) 2019-05-09
JP2018511588A (ja) 2018-04-26
JP2021127347A (ja) 2021-09-02
US11090368B2 (en) 2021-08-17
US20240009281A1 (en) 2024-01-11
AU2024202731B2 (en) 2026-04-23
JP6895892B2 (ja) 2021-06-30
AU2021232818B2 (en) 2024-02-01
JP2023126977A (ja) 2023-09-12
EP3270894A1 (en) 2018-01-24
US10172924B2 (en) 2019-01-08
AU2016233135A1 (en) 2017-09-28
EP3270894B1 (en) 2021-02-24
EP3900702A1 (en) 2021-10-27
WO2016149508A1 (en) 2016-09-22

Similar Documents

Publication Publication Date Title
AU2024202731B2 (en) Mrna therapy for pompe disease
US12533402B2 (en) MRNA therapy for argininosuccinate synthetase deficiency
US12509666B2 (en) MRNA therapy for phenylketonuria
US12201677B2 (en) Messenger RNA therapy for the treatment of ornithine transcarbamylase deficiency
HK40103984A (en) Mrna therapy for argininosuccinate synthetase deficiency

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20210315

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 8TH ANNIV.) - STANDARD

Year of fee payment: 8

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20240912

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20240912

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20240912

Q17 Modified document published

Free format text: ST27 STATUS EVENT CODE: A-4-4-Q10-Q17-Q103 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DOCUMENT PUBLISHED

Effective date: 20250130

F11 Ip right granted following substantive examination

Free format text: ST27 STATUS EVENT CODE: A-4-4-F10-F11-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: GRANT BY ISSUANCE

Effective date: 20250515

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-4-4-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20251203

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-4-4-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20251209

H13 Ip right lapsed

Free format text: ST27 STATUS EVENT CODE: N-4-6-H10-H13-H100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE AND LATE FEE NOT PAID BY DEADLINE OF NOTICE

Effective date: 20260317