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

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

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JP7114485B2
JP7114485B2 JP2018560595A JP2018560595A JP7114485B2 JP 7114485 B2 JP7114485 B2 JP 7114485B2 JP 2018560595 A JP2018560595 A JP 2018560595A JP 2018560595 A JP2018560595 A JP 2018560595A JP 7114485 B2 JP7114485 B2 JP 7114485B2
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gla
alkyl
alkenyl
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mrna
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パオロ マルティーニ，
スティーブン ホッジ，
ケリー ベネナート，
ウラジミール プレスニャク，
イアン マクファディン，
エララヘワーゲ サティヤジト クマーラシンヘ，
スーリン チュー，
リン タン グエイ，
ステーシ サブニス，
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ModernaTx Inc
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    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
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    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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JP2018560595A 2016-05-18 2017-05-18 ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド Active JP7114485B2 (ja)

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

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

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JP2019516719A5 JP2019516719A5 (https=) 2020-06-25
JP7114485B2 true JP7114485B2 (ja) 2022-08-08

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

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Cited By (1)

* Cited by examiner, † Cited by third party
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JP2022031359A (ja) * 2015-12-10 2022-02-18 モデルナティエックス インコーポレイテッド 治療薬の送達のための組成物及び方法

Families Citing this family (87)

* 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
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
FI3237621T3 (fi) 2014-12-22 2023-06-01 Codexis Inc Ihmisen alfa-galaktosidaasivariantteja
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
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
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
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)
EP3364981A4 (en) 2015-10-22 2019-08-07 ModernaTX, Inc. VACCINE AGAINST THE HUMAN CYTOMEGALOVIRUS
EP3364949A4 (en) 2015-10-22 2019-07-31 ModernaTX, Inc. CANCER VACCINES
TW201729835A (zh) 2015-10-22 2017-09-01 現代公司 呼吸道病毒疫苗
HUE057877T2 (hu) 2015-12-22 2022-06-28 Modernatx Inc Vegyületek és készítmények terápiás szerek intracelluláris bejuttatására
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
MA45052A (fr) 2016-05-18 2019-03-27 Modernatx Inc Polynucléotides codant pour jagged1 pour le traitement du syndrome d'alagille
AU2017266932B2 (en) 2016-05-18 2023-04-20 Modernatx, Inc. Polynucleotides encoding alpha-galactosidase A for the treatment of Fabry disease
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
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
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
WO2018170322A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Crystal forms of amino lipids
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
US11752206B2 (en) 2017-03-15 2023-09-12 Modernatx, Inc. Herpes simplex virus vaccine
US11969506B2 (en) 2017-03-15 2024-04-30 Modernatx, Inc. Lipid nanoparticle formulation
MA47790A (fr) 2017-03-17 2021-05-05 Modernatx Inc Vaccins à base d'arn contre des maladies zoonotiques
EP3601580A4 (en) * 2017-03-23 2021-01-06 Medicago Inc. NOROVIRUS FUSION PROTEINS AND VLPS WITH NOROVIRUS FUSION PROTEINS
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
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
WO2019036683A1 (en) 2017-08-18 2019-02-21 Modernatx, Inc. ANALYTICAL METHODS BY HPLC
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
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
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
EP3759154A1 (en) 2018-03-02 2021-01-06 Sigilon Therapeutics, Inc. Biocompatible hydrogel capsules and process for preparing same
JP2021516271A (ja) 2018-03-02 2021-07-01 シギロン セラピューティクス， インコーポレイテッドＳｉｇｉｌｏｎ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． 抗線維化化合物、デバイス、及びその使用
IL312315A (en) 2018-04-04 2024-06-01 Sigilon Therapeutics Inc Implantable particles and related methods
AU2019345067A1 (en) 2018-09-19 2021-04-08 Modernatx, Inc. High-purity peg lipids and uses thereof
MA53650A (fr) 2018-09-19 2021-07-28 Modernatx Inc Lipides peg et leurs utilisations
US12263248B2 (en) 2018-09-19 2025-04-01 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
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
SG11202106348QA (en) 2018-12-20 2021-07-29 Codexis Inc Human alpha-galactosidase variants
JP7716338B2 (ja) * 2019-01-04 2025-07-31 サンガモ セラピューティクス， インコーポレイテッド ファブリー病の処置のための方法及び組成物
US11351242B1 (en) 2019-02-12 2022-06-07 Modernatx, Inc. HMPV/hPIV3 mRNA vaccine composition
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
US11851694B1 (en) 2019-02-20 2023-12-26 Modernatx, Inc. High fidelity in vitro transcription
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
CN113840620A (zh) * 2019-03-27 2021-12-24 西吉隆医疗股份有限公司 用于治疗法布里病的组合物、装置和方法
EP3965830A1 (en) * 2019-05-07 2022-03-16 ModernaTX, Inc. Differentially expressed immune cell micrornas for regulation of protein expression
CA3150061A1 (en) * 2019-08-07 2021-02-11 Modernatx, Inc. Compositions and methods for enhanced delivery of agents
BR112022004936A2 (pt) 2019-09-19 2022-06-14 Modernatx Inc Compostos lipídicos contendo carbonato e composições para distribuição intracelular de agentes terapêuticos
AU2020350759A1 (en) 2019-09-19 2022-03-31 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
TW202146648A (zh) * 2020-02-28 2021-12-16 美商克迪科思股份有限公司 人類α-半乳糖苷酶變異體
WO2021183895A1 (en) * 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
EP4132576A1 (en) 2020-04-09 2023-02-15 Suzhou Abogen Biosciences Co., Ltd. Nucleic acid vaccines for coronavirus
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
KR102314642B1 (ko) * 2020-04-17 2021-10-20 재단법인 아산사회복지재단 파브리 병 진단용 바이오 마커 및 이의 용도
EP3901261A1 (en) 2020-04-22 2021-10-27 BioNTech RNA Pharmaceuticals GmbH Coronavirus vaccine
AU2021263563B2 (en) * 2020-04-27 2023-02-02 4D Molecular Therapeutics Inc. Codon optimized GLA genes and uses thereof
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
EP4277988A4 (en) * 2021-01-14 2024-12-11 Spark Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF FABRY'S DISEASE
NL2027654B1 (en) * 2021-02-08 2022-09-09 Nutcracker Therapeutics Inc Methods for manufacturing a syntetic template
US20220363937A1 (en) 2021-05-14 2022-11-17 Armstrong World Industries, Inc. Stabilization of antimicrobial coatings
US12186387B2 (en) 2021-11-29 2025-01-07 BioNTech SE Coronavirus vaccine
US20230340077A1 (en) * 2021-12-01 2023-10-26 Luyef Biotechnologies Inc. Production of myoglobin from trichoderma using a feeding media
US12529047B1 (en) 2021-12-21 2026-01-20 Modernatx, Inc. mRNA quantification methods
US20250099392A1 (en) * 2022-01-20 2025-03-27 Ohio State Innovation Foundation Compositions comprising lipid compounds and methods of making and use thereof
CN114672501B (zh) * 2022-05-27 2022-08-23 珠海丽凡达生物技术有限公司 一种mRNA、药物组合物及其应用
WO2024002985A1 (en) 2022-06-26 2024-01-04 BioNTech SE Coronavirus vaccine
CN121293183B (zh) * 2025-12-15 2026-03-24 北京悦康科创医药科技股份有限公司 含有苯并噻吩结构用于激活sting通路的佐剂脂质及其组合物

Citations (4)

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

Family Cites Families (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5082767A (en) 1989-02-27 1992-01-21 Hatfield G Wesley Codon pair utilization
WO2000018778A1 (en) 1998-09-29 2000-04-06 Phylos, Inc. Synthesis of codon randomized nucleic acids
US20090148906A1 (en) 1998-09-29 2009-06-11 Shire Human Genetic Therapies, Inc. A Delaware Corporation Optimized messenger rna
AR034853A1 (es) 2000-10-11 2004-03-24 Transkaryotic Therapies Inc Secuencia de acido nucleico sintetico que codifica una alfa-galactosidasa, vector y celula que la comprenden, metodo para producir alfa-galactosidasa, metodo para prepararla, composicion farmaceutica que comprende dicha secuencia y su uso para preparar esta ultima
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
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
US7683036B2 (en) 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
US8101385B2 (en) 2005-06-30 2012-01-24 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
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
WO2009030254A1 (en) 2007-09-04 2009-03-12 Curevac Gmbh Complexes of rna and cationic peptides for transfection and for immunostimulation
DK2205618T3 (en) 2007-09-26 2017-02-20 Intrexon Corp SYNTHETIC 5 'NON-TRANSLATED REGIONS, EXPRESSION VECTORS AND PROCEDURES FOR INCREASING TRANSGENIC EXPRESSION
WO2009075886A1 (en) 2007-12-11 2009-06-18 The Scripps Research Institute Compositions and methods related to mrna translational enhancer elements
US8401798B2 (en) 2008-06-06 2013-03-19 Dna Twopointo, Inc. Systems and methods for constructing frequency lookup tables for expression systems
US8126653B2 (en) 2008-07-31 2012-02-28 Dna Twopointo, Inc. Synthetic nucleic acids for expression of encoded proteins
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
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
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
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
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
EP3354644A1 (en) 2011-06-08 2018-08-01 Translate Bio, Inc. Cleavable lipids
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
CA2863964C (en) 2012-02-07 2021-10-26 Global Bio Therapeutics Usa, Inc. Compartmentalized method of nucleic acid delivery and compositions and uses thereof
WO2013120497A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
ES2762873T3 (es) 2012-03-29 2020-05-26 Translate Bio Inc Lípidos catiónicos ionizables
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
US10245229B2 (en) 2012-06-08 2019-04-02 Translate Bio, Inc. Pulmonary delivery of mRNA to non-lung target cells
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
EP4331620A3 (en) 2012-12-07 2024-12-04 Translate Bio, Inc. Lipidic nanoparticles for mrna delivery
US20140200261A1 (en) * 2013-01-17 2014-07-17 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
SG11201507474QA (en) 2013-03-14 2015-10-29 Shire Human Genetic Therapies RIBONUCLEIC ACIDs WITH 4'-THIO-MODIFIED NUCLEOTIDES AND RELATED METHODS
CA2906732C (en) * 2013-03-15 2023-08-08 The University Of British Columbia Lipid nanoparticles for transfection and related methods
US10130649B2 (en) 2013-03-15 2018-11-20 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
US10023626B2 (en) * 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
TW202332774A (zh) * 2013-10-23 2023-08-16 美商健臻公司 重組醣蛋白及其用途
US10821175B2 (en) 2014-02-25 2020-11-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
SG10201912038TA (en) * 2014-04-23 2020-02-27 Modernatx Inc Nucleic acid vaccines
US20180000953A1 (en) * 2015-01-21 2018-01-04 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
EP4219532A3 (en) 2015-06-05 2023-08-16 Dana-Farber Cancer Institute, Inc. Compositions and methods for transient gene therapy with enhanced stability
EP4286012A3 (en) * 2015-09-17 2024-05-29 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
US11377656B2 (en) 2016-03-10 2022-07-05 Novartis Ag Chemically modified messenger RNA's
EP3452101A2 (en) 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
AU2017266932B2 (en) 2016-05-18 2023-04-20 Modernatx, Inc. Polynucleotides encoding alpha-galactosidase A for the treatment of Fabry disease
MA46761A (fr) 2016-11-10 2019-09-18 Translate Bio Inc Administration sous-cutanée d'arn messager

Patent Citations (5)

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

Non-Patent Citations (1)

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

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022031359A (ja) * 2015-12-10 2022-02-18 モデルナティエックス インコーポレイテッド 治療薬の送達のための組成物及び方法
JP7412840B2 (ja) 2015-12-10 2024-01-15 モデルナティエックス インコーポレイテッド 治療薬の送達のための組成物及び方法

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