JP2019519511A5 - - Google Patents

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JP2019519511A5
JP2019519511A5 JP2018560776A JP2018560776A JP2019519511A5 JP 2019519511 A5 JP2019519511 A5 JP 2019519511A5 JP 2018560776 A JP2018560776 A JP 2018560776A JP 2018560776 A JP2018560776 A JP 2018560776A JP 2019519511 A5 JP2019519511 A5 JP 2019519511A5
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hours
glycero
mrna
pharmaceutical composition
phosphocholine
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JP2018560776A
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JP7210287B2 (ja
JP2019519511A (ja
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JP2018560776A 2016-05-18 2017-05-18 Ii型シトルリン血症の治療のためのシトリンをコードするポリヌクレオチド Active JP7210287B2 (ja)

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Application Number Priority Date Filing Date Title
JP2023002608A JP2023055716A (ja) 2016-05-18 2023-01-11 Ii型シトルリン血症の治療のためのシトリンをコードするポリヌクレオチド

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US201662338479P 2016-05-18 2016-05-18
US62/338,479 2016-05-18
PCT/US2017/033421 WO2017201349A1 (en) 2016-05-18 2017-05-18 Polynucleotides encoding citrin for the treatment of citrullinemia type 2

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JP2019519511A JP2019519511A (ja) 2019-07-11
JP2019519511A5 true JP2019519511A5 (https=) 2020-07-02
JP7210287B2 JP7210287B2 (ja) 2023-01-23

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JP2023002608A Pending JP2023055716A (ja) 2016-05-18 2023-01-11 Ii型シトルリン血症の治療のためのシトリンをコードするポリヌクレオチド

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US (2) US10993918B2 (https=)
EP (1) EP3458081B1 (https=)
JP (2) JP7210287B2 (https=)
AU (2) AU2017268396B2 (https=)
CA (1) CA3024625A1 (https=)
MA (1) MA45036A (https=)
MY (1) MY201498A (https=)
SG (1) SG11201810162PA (https=)
WO (1) WO2017201349A1 (https=)

Families Citing this family (100)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
EP3027221A1 (en) 2013-07-30 2016-06-08 PhaseRx, Inc. Block copolymers and their conjugates or complexes with oligonucleotides
WO2015105955A1 (en) 2014-01-08 2015-07-16 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
SG10201912038TA (en) 2014-04-23 2020-02-27 Modernatx Inc Nucleic acid vaccines
US11219634B2 (en) 2015-01-21 2022-01-11 Genevant Sciences Gmbh Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
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
EP3324979B1 (en) 2015-07-21 2022-10-12 ModernaTX, Inc. Infectious disease vaccines
US11364292B2 (en) 2015-07-21 2022-06-21 Modernatx, Inc. CHIKV RNA vaccines
US11474085B2 (en) 2015-07-28 2022-10-18 Flodesign Sonics, Inc. Expanded bed affinity selection
US11459540B2 (en) 2015-07-28 2022-10-04 Flodesign Sonics, Inc. Expanded bed affinity selection
WO2017020026A1 (en) 2015-07-30 2017-02-02 Modernatx, Inc. Concatemeric peptide epitopes rnas
WO2017031232A1 (en) 2015-08-17 2017-02-23 Modernatx, Inc. Methods for preparing particles and related compositions
EP4286012A3 (en) 2015-09-17 2024-05-29 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
EP3364981A4 (en) 2015-10-22 2019-08-07 ModernaTX, Inc. VACCINE AGAINST THE HUMAN CYTOMEGALOVIRUS
EP3364950A4 (en) 2015-10-22 2019-10-23 ModernaTX, Inc. VACCINES AGAINST TROPICAL DISEASES
CA3002912A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Nucleic acid vaccines for varicella zoster virus (vzv)
TW201729835A (zh) 2015-10-22 2017-09-01 現代公司 呼吸道病毒疫苗
EP3364949A4 (en) 2015-10-22 2019-07-31 ModernaTX, Inc. CANCER VACCINES
IL307179A (en) 2015-10-28 2023-11-01 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
HRP20220652T1 (hr) * 2015-12-10 2022-06-24 Modernatx, Inc. Pripravci i postupci unosa terapijskih sredstava
MA43568A (fr) 2015-12-17 2018-11-14 Modernatx Inc Polynucléotides codant pour la méthylmalonyl-coa mutase
HUE057877T2 (hu) 2015-12-22 2022-06-28 Modernatx Inc Vegyületek és készítmények terápiás szerek intracelluláris bejuttatására
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
MA45032A (fr) 2016-05-18 2019-03-27 Fundacion Para La Investig Medica Aplicada Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë
EP3458106A4 (en) 2016-05-18 2020-03-18 Modernatx, Inc. POLYNUCLEOTIDS FOR CODING LIPOPROTEIN LIPASE FOR TREATING HYPERLIPIDEMIA
MA45052A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour jagged1 pour le traitement du syndrome d'alagille
WO2017201347A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
WO2017201348A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
CN116837052A (zh) 2016-09-14 2023-10-03 摩登纳特斯有限公司 高纯度rna组合物及其制备方法
CA3041517A1 (en) 2016-10-19 2018-04-26 Flodesign Sonics, Inc. Affinity cell extraction by acoustics
MA46584A (fr) 2016-10-21 2019-08-28 Modernatx Inc Vaccin contre le cytomégalovirus humain
WO2018089851A2 (en) 2016-11-11 2018-05-17 Modernatx, Inc. Influenza vaccine
WO2018107088A2 (en) 2016-12-08 2018-06-14 Modernatx, Inc. Respiratory virus nucleic acid vaccines
WO2018111967A1 (en) 2016-12-13 2018-06-21 Modernatx, Inc. Rna affinity purification
WO2018126084A1 (en) 2016-12-30 2018-07-05 Phaserx, Inc. Branched peg molecules and related compositions and methods
US11045540B2 (en) 2017-03-15 2021-06-29 Modernatx, Inc. Varicella zoster virus (VZV) vaccine
WO2018170245A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Broad spectrum influenza virus vaccine
US11969506B2 (en) 2017-03-15 2024-04-30 Modernatx, Inc. Lipid nanoparticle formulation
WO2018170322A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Crystal forms of amino lipids
US11752206B2 (en) * 2017-03-15 2023-09-12 Modernatx, Inc. Herpes simplex virus vaccine
SMT202300097T1 (it) 2017-03-15 2023-05-12 Modernatx Inc Composto e composizioni per il rilascio intracellulare di agenti terapeutici
EP3595713A4 (en) 2017-03-15 2021-01-13 ModernaTX, Inc. RESPIRATORY SYNCYTIAL VIRUS Vaccine
MA47790A (fr) 2017-03-17 2021-05-05 Modernatx Inc Vaccins à base d'arn contre des maladies zoonotiques
AU2018240515B2 (en) 2017-03-24 2024-07-25 CureVac SE Nucleic acids encoding CRISPR-associated proteins and uses thereof
WO2018187590A1 (en) 2017-04-05 2018-10-11 Modernatx, Inc. Reduction or elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins
WO2018191657A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
US12077501B2 (en) 2017-06-14 2024-09-03 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
US11786607B2 (en) 2017-06-15 2023-10-17 Modernatx, Inc. RNA formulations
WO2019036685A1 (en) 2017-08-18 2019-02-21 Modernatx, Inc. METHODS FOR HPLC ANALYSIS
ES2983060T3 (es) 2017-08-18 2024-10-21 Modernatx Inc Variantes de ARN polimerasa
WO2019036683A1 (en) 2017-08-18 2019-02-21 Modernatx, Inc. ANALYTICAL METHODS BY HPLC
WO2019046809A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. METHODS OF MANUFACTURING LIPID NANOPARTICLES
EP3681514A4 (en) 2017-09-14 2021-07-14 ModernaTX, Inc. RNA VACZINE AGAINST ZIKA VIRUS
EP3714048B1 (en) 2017-11-22 2025-04-09 ModernaTX, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
US20220265856A1 (en) * 2017-11-22 2022-08-25 Modernatx, Inc. Polynucleotides encoding propionyl-coa carboxylase alpha and beta subunits for the treatment of propionic acidemia
JP7423521B2 (ja) * 2017-11-22 2024-01-29 モダーナティエックス・インコーポレイテッド フェニルケトン尿症の治療用のフェニルアラニンヒドロキシラーゼをコードするポリヌクレオチド
WO2019118921A1 (en) 2017-12-14 2019-06-20 Flodesign Sonics, Inc. Acoustic transducer drive and controller
CN111712468A (zh) 2017-12-22 2020-09-25 北卡罗莱纳州立大学 聚合物荧光团、包含其的组合物及制备和使用其的方法
WO2019148101A1 (en) 2018-01-29 2019-08-01 Modernatx, Inc. Rsv rna vaccines
JP2021512090A (ja) * 2018-01-30 2021-05-13 モデルナティーエックス, インコーポレイテッド 免疫細胞に薬剤を送達するための組成物及び方法
WO2019176079A1 (ja) * 2018-03-16 2019-09-19 株式会社 東芝 生分解性化合物、脂質粒子、脂質粒子を含む組成物、およびキット
CA3100276A1 (en) 2018-06-06 2019-12-12 Massachusetts Institute Of Technology Circular rna for translation in eukaryotic cells
AU2019345067A1 (en) 2018-09-19 2021-04-08 Modernatx, Inc. High-purity peg lipids and uses thereof
US12263248B2 (en) 2018-09-19 2025-04-01 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
MA53650A (fr) 2018-09-19 2021-07-28 Modernatx Inc Lipides peg et leurs utilisations
CN113271926A (zh) 2018-09-20 2021-08-17 摩登纳特斯有限公司 脂质纳米颗粒的制备及其施用方法
JP7543259B2 (ja) 2018-10-18 2024-09-02 アクイタス セラピューティクス インコーポレイテッド 活性剤の脂質ナノ粒子送達のための脂質
US11351242B1 (en) 2019-02-12 2022-06-07 Modernatx, Inc. HMPV/hPIV3 mRNA vaccine composition
US11851694B1 (en) 2019-02-20 2023-12-26 Modernatx, Inc. High fidelity in vitro transcription
CN113795579A (zh) 2019-02-20 2021-12-14 摩登纳特斯有限公司 用于共转录加帽的rna聚合酶变体
CA3132975A1 (en) 2019-03-11 2020-09-17 Modernatx, Inc. Fed-batch in vitro transcription process
EP3938379A4 (en) 2019-03-15 2023-02-22 ModernaTX, Inc. Hiv rna vaccines
WO2020237227A1 (en) 2019-05-22 2020-11-26 Massachusetts Institute Of Technology Circular rna compositions and methods
AU2020350759A1 (en) 2019-09-19 2022-03-31 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
BR112022004936A2 (pt) 2019-09-19 2022-06-14 Modernatx Inc Compostos lipídicos contendo carbonato e composições para distribuição intracelular de agentes terapêuticos
AU2020397956A1 (en) 2019-12-04 2022-07-07 Orna Therapeutics, Inc. Circular RNA compositions and methods
JP7681617B2 (ja) 2020-04-09 2025-05-22 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
EP4132576A1 (en) 2020-04-09 2023-02-15 Suzhou Abogen Biosciences Co., Ltd. Nucleic acid vaccines for coronavirus
EP3901261A1 (en) 2020-04-22 2021-10-27 BioNTech RNA Pharmaceuticals GmbH Coronavirus vaccine
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
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
US12329811B2 (en) 2021-01-11 2025-06-17 Modernatx, Inc. Seasonal RNA influenza virus vaccines
CN114763324A (zh) * 2021-01-15 2022-07-19 纳肽得(青岛)生物医药有限公司 一种脂质化合物及脂质体与药物组合物
US20220363937A1 (en) 2021-05-14 2022-11-17 Armstrong World Industries, Inc. Stabilization of antimicrobial coatings
WO2022266032A1 (en) * 2021-06-14 2022-12-22 Generation Bio Co. Cationic lipids and compositions thereof
US12186387B2 (en) 2021-11-29 2025-01-07 BioNTech SE Coronavirus vaccine
US12529047B1 (en) 2021-12-21 2026-01-20 Modernatx, Inc. mRNA quantification methods
TW202330457A (zh) * 2022-01-19 2023-08-01 大陸商蘇州艾博生物科技有限公司 脂質化合物和脂質奈米顆粒組合物
WO2023222081A1 (zh) 2022-05-19 2023-11-23 仁景(苏州)生物科技有限公司 长链烷基酯胺类脂质化合物及其制备方法和在核酸递送方面的应用
US12297285B2 (en) 2022-06-24 2025-05-13 Orna Therapeutics, Inc. Circular RNA encoding chimeric antigen receptors targeting BCMA
WO2024002985A1 (en) 2022-06-26 2024-01-04 BioNTech SE Coronavirus vaccine
CN114875033A (zh) * 2022-06-29 2022-08-09 福建省医学科学研究院 sgRNA、CRISPR/Cas试剂及其应用
CN116535381B (zh) * 2023-07-06 2023-10-17 北京悦康科创医药科技股份有限公司 具有五元环缩醛结构的阳离子脂质化合物、包含其的组合物及用途

Family Cites Families (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5082767A (en) 1989-02-27 1992-01-21 Hatfield G Wesley Codon pair utilization
WO2000018778A1 (en) 1998-09-29 2000-04-06 Phylos, Inc. Synthesis of codon randomized nucleic acids
JP4480199B2 (ja) 1999-05-10 2010-06-16 圭子 小林 成人発症ii型シトルリン血症の診断方法
ATE490267T1 (de) 2001-06-05 2010-12-15 Curevac Gmbh Stabilisierte mrna mit erhöhtem g/c-gehalt, kodierend für ein virales antigen
EP2390331B1 (en) 2001-09-28 2018-04-25 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MicroRNA molecules
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
US20040005600A1 (en) 2002-04-01 2004-01-08 Evelina Angov Method of designing synthetic nucleic acid sequences for optimal protein expression in a host cell
DE10260805A1 (de) 2002-12-23 2004-07-22 Geneart Gmbh Verfahren und Vorrichtung zum Optimieren einer Nucleotidsequenz zur Expression eines Proteins
JP2004315469A (ja) 2003-04-18 2004-11-11 Ajinomoto Co Inc シトルリン血症治療剤
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
JP2006001868A (ja) 2004-06-16 2006-01-05 Ajinomoto Co Inc シトルリン血症治療剤
JP4912574B2 (ja) 2004-07-22 2012-04-11 味の素株式会社 シトルリン血症治療剤
US8101385B2 (en) 2005-06-30 2012-01-24 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
KR20080025181A (ko) 2005-06-30 2008-03-19 아케믹스 코포레이션 완전 2'-변형된 핵산 전사체를 생성하기 위한 물질 및 방법
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
US20080046192A1 (en) 2006-08-16 2008-02-21 Richard Lathrop Polypepetide-encoding nucleotide sequences with refined translational kinetics and methods of making same
WO2008078180A2 (en) 2006-12-22 2008-07-03 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
DK2205618T3 (en) 2007-09-26 2017-02-20 Intrexon Corp SYNTHETIC 5 'NON-TRANSLATED REGIONS, EXPRESSION VECTORS AND PROCEDURES FOR INCREASING TRANSGENIC EXPRESSION
WO2009075886A1 (en) 2007-12-11 2009-06-18 The Scripps Research Institute Compositions and methods related to mrna translational enhancer elements
US7561973B1 (en) 2008-07-31 2009-07-14 Dna Twopointo, Inc. Methods for determining properties that affect an expression property value of polynucleotides in an expression system
US8401798B2 (en) 2008-06-06 2013-03-19 Dna Twopointo, Inc. Systems and methods for constructing frequency lookup tables for expression systems
PL215513B1 (pl) 2008-06-06 2013-12-31 Univ Warszawski Nowe boranofosforanowe analogi dinukleotydów, ich zastosowanie, czasteczka RNA, sposób otrzymywania RNA oraz sposób otrzymywania peptydów lub bialka
US8126653B2 (en) 2008-07-31 2012-02-28 Dna Twopointo, Inc. Synthetic nucleic acids for expression of encoded proteins
MX342785B (es) 2009-06-10 2016-10-12 Alnylam Pharmaceuticals Inc Formulacion mejorada de lipido.
US20110082055A1 (en) 2009-09-18 2011-04-07 Codexis, Inc. Reduced codon mutagenesis
HUE042177T2 (hu) * 2009-12-01 2019-06-28 Translate Bio Inc Szteroidszármazék mRNS szállítására humán genetikai betegségekben
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
EP2691101A2 (en) 2011-03-31 2014-02-05 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
NZ616048A (en) 2011-04-26 2015-07-31 Vectura Gmbh Administration of iloprost as aerosol bolus
EP2755986A4 (en) 2011-09-12 2015-05-20 Moderna Therapeutics Inc MANIPULATED NUCLEIC ACIDS AND METHOD OF APPLICATION THEREFOR
RU2648950C2 (ru) 2011-10-03 2018-04-02 Модерна Терапьютикс, Инк. Модифицированные нуклеозиды, нуклеотиды и нуклеиновые кислоты и их применение
US20140308304A1 (en) 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
CA2868391A1 (en) 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same
US9512456B2 (en) 2012-08-14 2016-12-06 Modernatx, Inc. Enzymes and polymerases for the synthesis of RNA
WO2014093924A1 (en) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
WO2014089212A1 (en) * 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
US20140200261A1 (en) 2013-01-17 2014-07-17 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
US20160022840A1 (en) 2013-03-09 2016-01-28 Moderna Therapeutics, Inc. Heterologous untranslated regions for mrna
US10130649B2 (en) 2013-03-15 2018-11-20 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
US10821175B2 (en) 2014-02-25 2020-11-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
SMT202200502T1 (it) * 2014-06-25 2023-01-13 Acuitas Therapeutics Inc Nuovi lipidi e formulazioni di nanoparticelle lipidiche per l'erogazione di acidi nucleici
EP4286012A3 (en) 2015-09-17 2024-05-29 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
IL307179A (en) 2015-10-28 2023-11-01 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
MA43568A (fr) 2015-12-17 2018-11-14 Modernatx Inc Polynucléotides codant pour la méthylmalonyl-coa mutase
MA45032A (fr) 2016-05-18 2019-03-27 Fundacion Para La Investig Medica Aplicada Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë
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
WO2017201348A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
EP3458106A4 (en) 2016-05-18 2020-03-18 Modernatx, Inc. POLYNUCLEOTIDS FOR CODING LIPOPROTEIN LIPASE FOR TREATING HYPERLIPIDEMIA
WO2017201347A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis

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