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

Info

Publication number
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
Authority
JP
Japan
Prior art keywords
gla
alkyl
alkenyl
pharmaceutical composition
mrna
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
JP2018560595A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019516719A (ja
JP2019516719A5 (enExample
Inventor
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，
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
ModernaTx Inc
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 JP2019516719A publication Critical patent/JP2019516719A/ja
Publication of JP2019516719A5 publication Critical patent/JP2019516719A5/ja
Priority to JP2022119527A priority Critical patent/JP2022136230A/ja
Application granted granted Critical
Publication of JP7114485B2 publication Critical patent/JP7114485B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules 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
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • 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
    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • 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)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
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)

Priority Applications (1)

Application Number Priority Date Filing Date Title
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

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022119527A Division 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

Publications (3)

Publication Number Publication Date
JP2019516719A JP2019516719A (ja) 2019-06-20
JP2019516719A5 JP2019516719A5 (enExample) 2020-06-25
JP7114485B2 true JP7114485B2 (ja) 2022-08-08

Family

ID=59021568

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018560595A Active JP7114485B2 (ja) 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
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

Family Applications After (1)

Application Number Title Priority Date Filing Date
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

Country Status (6)

Country Link
US (5) US10519455B2 (enExample)
EP (2) EP3896164A1 (enExample)
JP (2) JP7114485B2 (enExample)
AU (1) AU2017266932B2 (enExample)
CA (1) CA3024507A1 (enExample)
WO (1) WO2017201328A1 (enExample)

Cited By (1)

* 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

Families Citing this family (82)

* 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
PT3981437T (pt) 2014-04-23 2025-01-15 Modernatx Inc Vacinas de ácidos nucleicos
US20170360900A1 (en) 2014-12-22 2017-12-21 Codexis, Inc. Human alpha-galactosidase variants
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
TW201718638A (zh) 2015-07-21 2017-06-01 現代治療公司 傳染病疫苗
EP3328394A4 (en) 2015-07-30 2019-03-13 ModernaTX, Inc. CONCATEMEE peptide epitope RNAs
US11564893B2 (en) 2015-08-17 2023-01-31 Modernatx, Inc. Methods for preparing particles and related compositions
SMT202200178T1 (it) 2015-09-17 2022-05-12 Modernatx Inc Composti e composizioni per il rilascio intracellulare di agenti terapeutici
EP3718565B1 (en) 2015-10-22 2022-04-27 ModernaTX, Inc. Respiratory virus vaccines
US11643441B1 (en) 2015-10-22 2023-05-09 Modernatx, Inc. Nucleic acid vaccines for varicella zoster virus (VZV)
JP6921833B2 (ja) 2015-10-22 2021-08-18 モデルナティーエックス， インコーポレイテッド ヒトサイトメガロウイルスワクチン
WO2017070624A1 (en) 2015-10-22 2017-04-27 Modernatx, Inc. Tropical disease vaccines
PL3394030T3 (pl) 2015-12-22 2022-04-11 Modernatx, Inc. Związki i kompozycje do wewnątrzkomórkowego dostarczania środków
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
EP3458107B1 (en) 2016-05-18 2024-03-13 ModernaTX, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
EP3896164A1 (en) 2016-05-18 2021-10-20 ModernaTX, Inc. Polynucleotides encoding alpha-galactosidase a for the treatment of fabry disease
CA3036831A1 (en) 2016-09-14 2018-03-22 Modernatx, Inc. High purity rna compositions and methods for preparation thereof
AU2017345766A1 (en) 2016-10-21 2019-05-16 Modernatx, Inc. Human cytomegalovirus vaccine
EP3538146A4 (en) 2016-11-11 2020-07-15 ModernaTX, Inc. INFLUENZA VACCINE
US11103578B2 (en) 2016-12-08 2021-08-31 Modernatx, Inc. Respiratory virus nucleic acid vaccines
US11384352B2 (en) 2016-12-13 2022-07-12 Modernatx, Inc. RNA affinity purification
WO2018170270A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Varicella zoster virus (vzv) vaccine
WO2018170256A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Herpes simplex virus vaccine
WO2018170245A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Broad spectrum influenza virus vaccine
AU2018234828A1 (en) 2017-03-15 2019-09-19 Modernatx, Inc. Lipid nanoparticle formulation
AU2018234814B2 (en) 2017-03-15 2022-06-30 Modernatx, Inc. Crystal forms of amino lipids
US11464848B2 (en) 2017-03-15 2022-10-11 Modernatx, Inc. Respiratory syncytial virus vaccine
LT3596041T (lt) 2017-03-15 2023-01-25 Modernatx, Inc. Terapinių medžiagų, skirtų intraląsteliniam tiekimui, junginys ir sudėtys
WO2018170347A1 (en) 2017-03-17 2018-09-20 Modernatx, Inc. Zoonotic disease rna vaccines
WO2018170603A1 (en) * 2017-03-23 2018-09-27 Medicago Inc. Norovirus fusion proteins and vlps comprising norovirus fusion proteins
EP3601576A1 (en) 2017-03-24 2020-02-05 CureVac AG Nucleic acids encoding crispr-associated proteins and uses thereof
MA48047A (fr) 2017-04-05 2020-02-12 Modernatx Inc Réduction ou élimination de réponses immunitaires à des protéines thérapeutiques administrées par voie non intraveineuse, par exemple par voie sous-cutanée
EP3638678B1 (en) 2017-06-14 2025-12-03 ModernaTX, Inc. Compounds and compositions for intracellular delivery of agents
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS
MA49914A (fr) 2017-08-18 2021-04-21 Modernatx Inc Procédés analytiques par hplc
WO2019036682A1 (en) 2017-08-18 2019-02-21 Modernatx, Inc. RNA VARIANTS POLYMERASE
US11912982B2 (en) 2017-08-18 2024-02-27 Modernatx, Inc. Methods for HPLC analysis
CN111315359A (zh) 2017-08-31 2020-06-19 摩登纳特斯有限公司 制备脂质纳米颗粒的方法
US10653767B2 (en) 2017-09-14 2020-05-19 Modernatx, Inc. Zika virus MRNA vaccines
BR112020009889A2 (pt) 2017-12-14 2020-11-03 Flodesign Sonics, Inc. acionador e controlador de transdutor acústico
EP3728137A4 (en) 2017-12-22 2021-12-08 North Carolina State University POLYMERIC FLUOROPHORES, COMPOSITIONS INCLUDING THEM, AND THEIR PREPARATION AND USE PROCESSES
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
WO2019169245A1 (en) 2018-03-02 2019-09-06 Sigilon Therapeutics, Inc. Biocompatible hydrogel capsules and process for preparing same
IL277713B2 (en) 2018-04-04 2024-09-01 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
CA3113436A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
EP3852732A1 (en) 2018-09-19 2021-07-28 ModernaTX, Inc. Peg lipids and uses thereof
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
BR112021011750A2 (pt) 2018-12-20 2021-08-31 Codexis, Inc. Alfa-galactosidase a recombinante e/ou fragmento de alfa-galactosidase a recombinante, composição, sequência de polinucleotídeo recombinante, vetor de expressão, célula hospedeira, métodos para produzir uma variante de alfa-galactosidase a e para tratar e/ou prevenir os sintomas da doença de fabry, composição farmacêutica, e, uso das composições
KR20210112337A (ko) * 2019-01-04 2021-09-14 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
US11351242B1 (en) 2019-02-12 2022-06-07 Modernatx, Inc. HMPV/hPIV3 mRNA vaccine composition
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
CN113874502A (zh) 2019-03-11 2021-12-31 摩登纳特斯有限公司 补料分批体外转录方法
WO2020190750A1 (en) 2019-03-15 2020-09-24 Modernatx, Inc. Hiv rna vaccines
US20220267794A1 (en) * 2019-03-27 2022-08-25 Sigilon Therapeutics, Inc. Compositions, devices, and methods for treating fabry disease
US20230086537A1 (en) * 2019-05-07 2023-03-23 Modernatx, Inc. Differentially expressed immune cell micrornas for regulation of protein expression
JP7766583B2 (ja) * 2019-08-07 2025-11-10 モデルナティエックス インコーポレイテッド 強化された薬剤送達のための組成物及び方法
BR112022004759A2 (pt) 2019-09-19 2022-06-21 Modernatx Inc Composições e compostos lipídicos com cauda ramificada para entrega intracelular de agentes terapêuticos
EP4110926A4 (en) * 2020-02-28 2024-06-19 Codexis, Inc. HUMAN ALPHA-GALACTOSIDASE VARIANTS
WO2021183895A1 (en) * 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
CN113874507A (zh) 2020-04-09 2021-12-31 苏州艾博生物科技有限公司 冠状病毒的核酸疫苗
CN117003658B (zh) 2020-04-09 2025-10-14 苏州艾博生物科技有限公司 脂质纳米颗粒组合物
KR102314642B1 (ko) * 2020-04-17 2021-10-20 재단법인 아산사회복지재단 파브리 병 진단용 바이오 마커 및 이의 용도
CA3174215A1 (en) 2020-04-22 2021-10-28 Ugur Sahin Coronavirus vaccine
TWI769795B (zh) 2020-04-27 2022-07-01 美商４Ｄ分子療法公司 密碼子優化的ｇｌａ基因及其用途
US11406703B2 (en) 2020-08-25 2022-08-09 Modernatx, Inc. Human cytomegalovirus vaccine
WO2022081750A1 (en) 2020-10-14 2022-04-21 George Mason Research Foundation, Inc. Ionizable lipids and methods of manufacture and use thereof
US12329811B2 (en) 2021-01-11 2025-06-17 Modernatx, Inc. Seasonal RNA influenza virus vaccines
JP2024504611A (ja) * 2021-01-14 2024-02-01 スパーク セラピューティクス インコーポレイテッド ファブリー病を治療するための組成物および方法
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
CN118891043A (zh) * 2022-01-20 2024-11-01 俄亥俄州创新基金会 包含脂质化合物的组合物以及制备和使用其的方法
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

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
US20090148906A1 (en) 1998-09-29 2009-06-11 Shire Human Genetic Therapies, Inc. A Delaware Corporation Optimized messenger rna
WO2000018778A1 (en) 1998-09-29 2000-04-06 Phylos, Inc. Synthesis of codon randomized nucleic acids
CA2425732A1 (en) 2000-10-11 2002-08-22 Transkaryotic Therapies, Inc. Optimized messenger rna
DE50214379D1 (de) 2001-06-05 2010-05-27 Curevac Gmbh Stabilisierte mRNA mit erhöhtem G/C-Gehalt für die Gentherapie
WO2003029459A2 (en) 2001-09-28 2003-04-10 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Micro-rna molecules
AU2003228440B2 (en) 2002-04-01 2008-10-02 Walter Reed Army Institute Of Research 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
EP1648914A4 (en) 2003-07-31 2009-12-16 Regulus Therapeutics Inc OLIGOMERIC COMPOUNDS AND COMPOSITIONS USEFUL FOR MODULATING SMALL NON-CODING RNA
KR20080025181A (ko) 2005-06-30 2008-03-19 아케믹스 코포레이션 완전 2'-변형된 핵산 전사체를 생성하기 위한 물질 및 방법
US8101385B2 (en) 2005-06-30 2012-01-24 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
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
AU2007337810B2 (en) 2006-12-22 2014-02-13 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
EP3156414B1 (en) 2007-09-26 2019-12-04 Intrexon Corporation Synthetic 5'utrs, expression vectors, and methods for increasing transgene expression
EP2584038B1 (en) 2007-12-11 2014-06-25 The Scripps Research Institute Compositions and methods related to mRNA translational enhancer elements
US8126653B2 (en) 2008-07-31 2012-02-28 Dna Twopointo, Inc. Synthetic nucleic acids for expression of encoded proteins
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
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
HRP20211619T1 (hr) 2009-06-10 2022-02-04 Arbutus Biopharma Corporation Poboljšana formulacija lipida
US20110082055A1 (en) 2009-09-18 2011-04-07 Codexis, Inc. Reduced codon mutagenesis
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
DK3202760T3 (da) 2011-01-11 2019-11-25 Alnylam Pharmaceuticals Inc Pegylerede lipider og deres anvendelse til lægemiddelfremføring
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
ES2987057T3 (es) 2011-06-08 2024-11-13 Translate Bio Inc Lípidos escindibles
WO2013039857A1 (en) 2011-09-12 2013-03-21 modeRNA Therapeutics Engineered nucleic acids and methods of use thereof
KR20190099538A (ko) 2011-10-03 2019-08-27 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드, 및 핵산, 및 이들의 용도
AU2013216920B2 (en) 2012-02-07 2016-09-22 Global Bio Therapeutics, 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
AU2013243954A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
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
US20150307542A1 (en) 2012-10-03 2015-10-29 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
JP2016504050A (ja) 2013-01-17 2016-02-12 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． 細胞表現型の改変のためのシグナルセンサーポリヌクレオチド
JP6567494B2 (ja) 2013-03-14 2019-08-28 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 ４’−チオ−修飾ヌクレオチドを有するリボ核酸及び関連方法
ES3032013T3 (en) 2013-03-15 2025-07-14 Translate Bio Inc Synergistic enhancement of the delivery of nucleic acids via blended formulations
EP2971013B1 (en) * 2013-03-15 2020-08-19 The University Of British Columbia Lipid nanoparticles for transfection and related methods
US10023626B2 (en) * 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
TWI642782B (zh) * 2013-10-23 2018-12-01 健臻公司 重組醣蛋白及其用途
EP3556353A3 (en) 2014-02-25 2020-03-18 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
PT3981437T (pt) * 2014-04-23 2025-01-15 Modernatx Inc Vacinas de ácidos nucleicos
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
EP3303377B1 (en) 2015-06-05 2022-11-23 Dana-Farber Cancer Institute, Inc. Compositions and methods for transient gene therapy with enhanced stability
SMT202200178T1 (it) 2015-09-17 2022-05-12 Modernatx Inc Composti e composizioni per il rilascio intracellulare di agenti terapeutici
US11377656B2 (en) 2016-03-10 2022-07-05 Novartis Ag Chemically modified messenger RNA's
US11078247B2 (en) 2016-05-04 2021-08-03 Curevac Ag RNA encoding a therapeutic protein
EP3896164A1 (en) 2016-05-18 2021-10-20 ModernaTX, Inc. Polynucleotides encoding alpha-galactosidase a for the treatment of fabry disease
WO2018089846A1 (en) 2016-11-10 2018-05-17 Translate Bio, Inc. Subcutaneous delivery of messenger rna

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

Also Published As

Publication number Publication date
EP3896164A1 (en) 2021-10-20
US20190002890A1 (en) 2019-01-03
US10494636B2 (en) 2019-12-03
WO2017201328A1 (en) 2017-11-23
AU2017266932A1 (en) 2018-11-29
AU2017266932B2 (en) 2023-04-20
EP3458590B9 (en) 2021-08-18
JP2019516719A (ja) 2019-06-20
US11649461B2 (en) 2023-05-16
JP2022136230A (ja) 2022-09-15
US20200149052A1 (en) 2020-05-14
CA3024507A1 (en) 2017-11-23
US20230323371A1 (en) 2023-10-12
AU2017266932A9 (en) 2019-08-29
EP3458590B1 (en) 2021-01-27
US10519455B2 (en) 2019-12-31
US20190000933A1 (en) 2019-01-03
US20190000932A1 (en) 2019-01-03
EP3458590A1 (en) 2019-03-27

Similar Documents

Publication Publication Date Title
JP7114485B2 (ja) 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
US12239742B2 (en) Polynucleotides encoding Citrin for the treatment of Citrullinemia type 2
US12377136B2 (en) Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria
US12252704B2 (en) Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
EP3458107B1 (en) Polynucleotides encoding jagged1 for the treatment of alagille syndrome
US20240318187A1 (en) Polyribonucleotides containing reduced uracil content and uses thereof
US20190298657A1 (en) Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency
AU2017266948A1 (en) Polynucleotides encoding porphobilinogen deaminase for the treatment of acute intermittent porphyria
HK40004218A (en) POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE
HK40004218B (en) POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE

Legal Events

Date Code Title Description
A524 Written submission of copy of amendment under article 19 pct

Free format text: JAPANESE INTERMEDIATE CODE: A524

Effective date: 20190204

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200518

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20200518

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210518

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20210813

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211018

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220317

RD03 Notification of appointment of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7423

Effective date: 20220516

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20220526

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220614

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20220628

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20220727

R150 Certificate of patent or registration of utility model

Ref document number: 7114485

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250