MA43568A - Polynucléotides codant pour la méthylmalonyl-coa mutase - Google Patents

Polynucléotides codant pour la méthylmalonyl-coa mutase

Info

Publication number
MA43568A
MA43568A MA043568A MA43568A MA43568A MA 43568 A MA43568 A MA 43568A MA 043568 A MA043568 A MA 043568A MA 43568 A MA43568 A MA 43568A MA 43568 A MA43568 A MA 43568A
Authority
MA
Morocco
Prior art keywords
methylmalonyl
coa mutase
polynucleotides coding
polynucleotides
coding
Prior art date
Application number
MA043568A
Other languages
English (en)
Inventor
Paolo Martini
Vladimir Presnyak
Original Assignee
Modernatx 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 Modernatx Inc filed Critical Modernatx Inc
Publication of MA43568A publication Critical patent/MA43568A/fr

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/52Isomerases (5)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Liposomes
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/5123Organic compounds, e.g. fats, sugars
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52Genes encoding for enzymes or proenzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/90Isomerases (5.)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y504/00Intramolecular transferases (5.4)
    • C12Y504/99Intramolecular transferases (5.4) transferring other groups (5.4.99)
    • C12Y504/99002Methylmalonyl-CoA mutase (5.4.99.2)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Dispersion Chemistry (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
MA043568A 2015-12-17 2016-12-16 Polynucléotides codant pour la méthylmalonyl-coa mutase MA43568A (fr)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201562269092P 2015-12-17 2015-12-17
US201562269089P 2015-12-17 2015-12-17
US201562273108P 2015-12-30 2015-12-30
US201562273112P 2015-12-30 2015-12-30
US201662274733P 2016-01-04 2016-01-04
US201662274726P 2016-01-04 2016-01-04
US201662274727P 2016-01-04 2016-01-04
US201662274722P 2016-01-04 2016-01-04
US201662338478P 2016-05-18 2016-05-18
US201662338456P 2016-05-18 2016-05-18
US201662409343P 2016-10-17 2016-10-17

Publications (1)

Publication Number Publication Date
MA43568A true MA43568A (fr) 2018-11-14

Family

ID=57738001

Family Applications (1)

Application Number Title Priority Date Filing Date
MA043568A MA43568A (fr) 2015-12-17 2016-12-16 Polynucléotides codant pour la méthylmalonyl-coa mutase

Country Status (8)

Country Link
US (5) US10426738B2 (fr)
EP (1) EP3390630A1 (fr)
JP (1) JP7065036B2 (fr)
AU (1) AU2016369612B2 (fr)
CA (1) CA3007108A1 (fr)
IL (2) IL259667B (fr)
MA (1) MA43568A (fr)
WO (1) WO2017106799A1 (fr)

Families Citing this family (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9725710B2 (en) 2014-01-08 2017-08-08 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
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
US11564893B2 (en) 2015-08-17 2023-01-31 Modernatx, Inc. Methods for preparing particles and related compositions
EP4349404A3 (fr) 2015-10-22 2024-06-19 ModernaTX, Inc. Vaccins contre le virus respiratoire
WO2017070613A1 (fr) 2015-10-22 2017-04-27 Modernatx, Inc. Vaccin contre le cytomégalovirus humain
EP3364950A4 (fr) 2015-10-22 2019-10-23 ModernaTX, Inc. Vaccins contre des maladies tropicales
EA201891001A1 (ru) 2015-10-22 2018-11-30 МОДЕРНАТиЭкс, ИНК. Вакцины на основе нуклеиновых кислот против вируса ветряной оспы (vzv)
DK3386484T3 (da) 2015-12-10 2022-07-04 Modernatx Inc Sammensætninger og fremgangsmåder til afgivelse af terapeutiske midler
CA3007108A1 (fr) 2015-12-17 2017-06-22 Modernatx, Inc. Polynucleotides codant pour la methylmalonyl-coa mutase
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
EP3458108A4 (fr) 2016-05-18 2020-04-22 ModernaTX, Inc. Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique
US11001861B2 (en) 2016-05-18 2021-05-11 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
US20190298657A1 (en) * 2016-05-18 2019-10-03 Modernatx, Inc. Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency
WO2017201349A1 (fr) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2
EP3512944B1 (fr) 2016-09-14 2022-07-06 ModernaTX, Inc. Compositions d'arn de haute pureté et procédés pour leur préparation
AU2017345766A1 (en) 2016-10-21 2019-05-16 Modernatx, Inc. Human cytomegalovirus vaccine
EP3538146A4 (fr) 2016-11-11 2020-07-15 ModernaTX, Inc. Vaccin antigrippal
MA50335A (fr) 2016-12-08 2020-08-19 Modernatx Inc Vaccins à acide nucléique contre des virus respiratoires
EP3555289A1 (fr) 2016-12-13 2019-10-23 ModernaTX, Inc. Purification par affinité d'arn
US11045540B2 (en) 2017-03-15 2021-06-29 Modernatx, Inc. Varicella zoster virus (VZV) vaccine
US11752206B2 (en) * 2017-03-15 2023-09-12 Modernatx, Inc. Herpes simplex virus vaccine
MA47787A (fr) 2017-03-15 2020-01-22 Modernatx Inc Vaccin contre le virus respiratoire syncytial
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
WO2018170245A1 (fr) 2017-03-15 2018-09-20 Modernatx, Inc. Vaccin à large spectre contre le virus de la grippe
EP3595676A4 (fr) 2017-03-17 2021-05-05 Modernatx, Inc. Vaccins à base d'arn contre des maladies zoonotiques
EP3607074A4 (fr) 2017-04-05 2021-07-07 Modernatx, Inc. Réduction ou élimination de réponses immunitaires à des protéines thérapeutiques administrées par voie non intraveineuse, par exemple par voie sous-cutanée
WO2018231990A2 (fr) * 2017-06-14 2018-12-20 Modernatx, Inc. Polynucléotides codant pour la méthylmalonyl-coa mutase
WO2018232357A1 (fr) 2017-06-15 2018-12-20 Modernatx, Inc. Formulations d'arn
MA49914A (fr) 2017-08-18 2021-04-21 Modernatx Inc Procédés analytiques par hplc
US11912982B2 (en) 2017-08-18 2024-02-27 Modernatx, Inc. Methods for HPLC analysis
CN111212905A (zh) 2017-08-18 2020-05-29 摩登纳特斯有限公司 Rna聚合酶变体
WO2019046809A1 (fr) 2017-08-31 2019-03-07 Modernatx, Inc. Procédés de fabrication de nanoparticules lipidiques
EP3679379A2 (fr) * 2017-09-08 2020-07-15 The U.S.A. as represented by the Secretary, Department of Health and Human Services Biomarqueurs d'acidémies organiques
WO2019055807A1 (fr) 2017-09-14 2019-03-21 Modernatx, Inc. Vaccins à arn contre le virus zika
AU2018385759B2 (en) 2017-12-14 2021-10-21 Flodesign Sonics, Inc. Acoustic transducer driver and controller
WO2019148101A1 (fr) 2018-01-29 2019-08-01 Modernatx, Inc. Vaccins à base d'arn contre le vrs
US20190314291A1 (en) * 2018-01-30 2019-10-17 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells
CN108704162A (zh) * 2018-08-08 2018-10-26 中国科学院长春应用化学研究所 一种吸水敷料及其制备方法
EP3849617A1 (fr) 2018-09-14 2021-07-21 Translate Bio, Inc. Compositions et méthodes de traitement de l'acidémie méthylmalonique
MX2021010075A (es) 2019-02-20 2021-12-10 Modernatx Inc Variantes de arn polimerasa para la formacion de casquetes cotranscripcionales.
US11851694B1 (en) 2019-02-20 2023-12-26 Modernatx, Inc. High fidelity in vitro transcription
US20220162278A1 (en) * 2019-02-27 2022-05-26 The Regents Of The University Of Michigan Carbamoyl phosphate synthatase-1 for the treatment and prevention of liver injury
WO2021204179A1 (fr) 2020-04-09 2021-10-14 Suzhou Abogen Biosciences Co., Ltd. Vaccins à base d'acide nucléique pour coronavirus
JP2023529522A (ja) 2020-04-09 2023-07-11 スージョウ・アボジェン・バイオサイエンシズ・カンパニー・リミテッド 脂質ナノ粒子組成物
BR112022019781A2 (pt) 2020-04-22 2022-12-13 BioNTech SE Vacina para o coronavírus
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
WO2022204369A1 (fr) * 2021-03-24 2022-09-29 Modernatx, Inc. Polynucléotides codant pour la méthylmalonyl-coa mutase pour le traitement de l'acidémie méthylmalonique
WO2022246020A1 (fr) * 2021-05-19 2022-11-24 Modernatx, Inc. Polynucléotides codant pour la méthylmalonyl-coa mutase pour le traitement de l'acidémie méthylmalonique
WO2022245980A1 (fr) * 2021-05-20 2022-11-24 City Of Hope Composés de mir-142 et leurs utilisations
AU2022336209A1 (en) 2021-09-03 2024-01-18 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
WO2023073228A1 (fr) 2021-10-29 2023-05-04 CureVac SE Arn circulaire amélioré pour exprimer des protéines thérapeutiques
WO2023144330A1 (fr) 2022-01-28 2023-08-03 CureVac SE Inhibiteurs de facteurs de transcription codés par un acide nucleique
WO2023227608A1 (fr) 2022-05-25 2023-11-30 Glaxosmithkline Biologicals Sa Vaccin à base d'acide nucléique codant pour un polypeptide antigénique fimh d'escherichia coli
US11878055B1 (en) 2022-06-26 2024-01-23 BioNTech SE Coronavirus vaccine
DE202023106198U1 (de) 2022-10-28 2024-03-21 CureVac SE Impfstoff auf Nukleinsäurebasis

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
AU695618B2 (en) 1994-04-11 1998-08-20 Baylor College Of Medicine Compositions and methods for gene therapy to treat disease
US6652886B2 (en) 2001-02-16 2003-11-25 Expression Genetics Biodegradable cationic copolymers of poly (alkylenimine) and poly (ethylene glycol) for the delivery of bioactive agents
US20070122885A1 (en) 2005-08-22 2007-05-31 Fermalogic, Inc. Methods of increasing production of secondary metabolites by manipulating metabolic pathways that include methylmalonyl-coa
ES2735531T3 (es) 2005-08-23 2019-12-19 Univ Pennsylvania ARN que contiene nucleósidos modificados y métodos de uso del mismo
WO2009030254A1 (fr) 2007-09-04 2009-03-12 Curevac Gmbh Complexes d'arn et de peptides cationiques pour transfection et immunostimulation
CA2816925C (fr) * 2009-11-04 2023-01-10 The University Of British Columbia Particules lipidiques contenant des acides nucleiques et procedes associes
ES2734973T3 (es) 2009-12-01 2019-12-13 Translate Bio Inc Administración de ARNm para el aumento de proteínas y enzimas en enfermedades genéticas humanas
SE535869C2 (sv) * 2010-04-01 2013-01-22 Elways Ab Ett för ett eller flera, elektriskt framdrivbara, fordon anpassat system (Laddningssystem).
US20140308304A1 (en) * 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
CN104428009A (zh) 2012-02-07 2015-03-18 全球生物疗法美国有限公司 核酸输送的区室化方法及其组合物和应用
EP3501550A1 (fr) 2012-04-02 2019-06-26 Moderna Therapeutics, Inc. Polynucléotides modifiés pour la production de protéines associées à une maladie humaine
CN104411338A (zh) * 2012-04-02 2015-03-11 现代治疗公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
JP2016500254A (ja) * 2012-12-05 2016-01-12 サンガモ バイオサイエンシーズ, インコーポレイテッド 代謝疾患の調節のための方法および組成物
EP2946014A2 (fr) * 2013-01-17 2015-11-25 Moderna Therapeutics, Inc. Polynucléotides capteurs de signal servant à modifier les phénotypes cellulaires
WO2014152513A1 (fr) 2013-03-14 2014-09-25 Shire Human Genetic Therapies, Inc. Acides ribonucléiques ayant des nucléotides 4'-thio-modifiés et procédés associés
WO2014151927A1 (fr) * 2013-03-14 2014-09-25 Ipg Photonics Corporation Connecteur de fibre optique universel
US9719080B2 (en) 2013-03-15 2017-08-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Synthetic methylmalonyl-CoA mutase transgene for the treatment of MUT class methylmalonic acidemia (MMA)
US9944918B2 (en) 2013-03-15 2018-04-17 The United States Of America, As Represented By The Secretary, Dept. Of Health & Human Services Synthetic methylmalonyl-CoA mutase transgene for the treatment of MUT class methylmalonic acidemia (MMA)
CA2919828C (fr) * 2013-07-30 2022-07-19 Phaserx, Inc. Copolymeres sequences et leur conjugues ou complexes avec des oligonucleotides
SI3766916T1 (sl) 2014-06-25 2023-01-31 Acuitas Therapeutics Inc. Formulacije novih lipidov in lipidnih nanodelcev za dostavo nukleinskih kislin
EP3041948B1 (fr) 2014-11-10 2019-01-09 Modernatx, Inc. Molécules d'acide nucléique de remplacement contenant une quantité réduite d'uracile et utilisations associées
CN114642735A (zh) 2015-01-21 2022-06-21 菲泽尔克斯公司 用于将治疗剂和诊断剂递送到细胞中的方法、组合物和系统
EP3285812A2 (fr) * 2015-04-24 2018-02-28 The United States of America, as represented by the Secretary, Department of Health and Human Services Forme marquée de l'enzyme mut, constructions génétiques comprenant celle-ci, et son utilisation en thérapie génique
PL3350157T3 (pl) 2015-09-17 2022-05-16 Modernatx, Inc. Związki i kompozycje do dostarczania wewnątrzkomórkowego środków terapeutycznych
FI3368507T3 (fi) 2015-10-28 2023-03-21 Acuitas Therapeutics Inc Uusia lipidejä ja lipidinanopartikkeliformulaatioita nukleiinihappojen annostelemiseksi
WO2017100551A1 (fr) 2015-12-09 2017-06-15 Alexion Pharmaceuticals, Inc. Séquences utr hétérologues pour l'amplification de l'expression d'arnm
CA3007108A1 (fr) 2015-12-17 2017-06-22 Modernatx, Inc. Polynucleotides codant pour la methylmalonyl-coa mutase
WO2017147720A1 (fr) 2016-03-04 2017-09-08 Exerkine Corporation Méthode de traitement d'un trouble du système nerveux central
US11377656B2 (en) 2016-03-10 2022-07-05 Novartis Ag Chemically modified messenger RNA's
EP3452101A2 (fr) 2016-05-04 2019-03-13 CureVac AG Arn codant pour une protéine thérapeutique
US20180126003A1 (en) 2016-05-04 2018-05-10 Curevac Ag New targets for rna therapeutics
EP3458106A4 (fr) 2016-05-18 2020-03-18 Modernatx, Inc. Polynucléotides codant pour la lipoprotéine lipase destinés au traitement de l'hyperlipidémie
US20190298657A1 (en) 2016-05-18 2019-10-03 Modernatx, Inc. Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency
WO2017201349A1 (fr) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2
JP2019519601A (ja) 2016-05-18 2019-07-11 モダーナティエックス・インコーポレイテッドModernaTX, Inc. 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド
EP3458108A4 (fr) 2016-05-18 2020-04-22 ModernaTX, Inc. Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique
US11001861B2 (en) 2016-05-18 2021-05-11 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
WO2018157009A1 (fr) 2017-02-24 2018-08-30 Modernatx, Inc. Thérapie de dystrophies musculaires à base d'acide nucléique
WO2018231990A2 (fr) 2017-06-14 2018-12-20 Modernatx, Inc. Polynucléotides codant pour la méthylmalonyl-coa mutase
EP3849617A1 (fr) 2018-09-14 2021-07-21 Translate Bio, Inc. Compositions et méthodes de traitement de l'acidémie méthylmalonique

Also Published As

Publication number Publication date
US20230270685A1 (en) 2023-08-31
JP7065036B2 (ja) 2022-05-11
IL289098A (en) 2022-02-01
WO2017106799A1 (fr) 2017-06-22
US11504337B2 (en) 2022-11-22
CA3007108A1 (fr) 2017-06-22
US10426738B2 (en) 2019-10-01
US20180271795A1 (en) 2018-09-27
EP3390630A1 (fr) 2018-10-24
US20190022019A1 (en) 2019-01-24
US10406112B2 (en) 2019-09-10
US20180289838A1 (en) 2018-10-11
JP2019500430A (ja) 2019-01-10
US20200078314A1 (en) 2020-03-12
AU2016369612A1 (en) 2018-06-14
IL259667A (en) 2018-07-31
IL259667B (en) 2022-01-01
AU2016369612B2 (en) 2023-06-01

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