JP7213238B2 - 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 - Google Patents

筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 Download PDF

Info

Publication number
JP7213238B2
JP7213238B2 JP2020522046A JP2020522046A JP7213238B2 JP 7213238 B2 JP7213238 B2 JP 7213238B2 JP 2020522046 A JP2020522046 A JP 2020522046A JP 2020522046 A JP2020522046 A JP 2020522046A JP 7213238 B2 JP7213238 B2 JP 7213238B2
Authority
JP
Japan
Prior art keywords
muscle
seq
microdystrophin
muscular dystrophy
nucleotide sequence
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
JP2020522046A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021500352A (ja
JP2021500352A5 (enExample
Inventor
ルイーズ ロディノ－カラパック，
ジェリー アール． メンデル，
Original Assignee
リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル
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 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル filed Critical リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル
Publication of JP2021500352A publication Critical patent/JP2021500352A/ja
Publication of JP2021500352A5 publication Critical patent/JP2021500352A5/ja
Application granted granted Critical
Publication of JP7213238B2 publication Critical patent/JP7213238B2/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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2020522046A 2017-10-18 2018-03-16 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 Active JP7213238B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762573955P 2017-10-18 2017-10-18
US62/573,955 2017-10-18
PCT/US2018/022853 WO2019078916A1 (en) 2017-10-18 2018-03-16 ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MUSCLE-SPECIFIC MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY

Publications (3)

Publication Number Publication Date
JP2021500352A JP2021500352A (ja) 2021-01-07
JP2021500352A5 JP2021500352A5 (enExample) 2021-04-30
JP7213238B2 true JP7213238B2 (ja) 2023-01-26

Family

ID=66173432

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020522046A Active JP7213238B2 (ja) 2017-10-18 2018-03-16 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達

Country Status (5)

Country Link
US (2) US11534501B2 (enExample)
EP (1) EP3697915A4 (enExample)
JP (1) JP7213238B2 (enExample)
MA (1) MA50836A (enExample)
WO (1) WO2019078916A1 (enExample)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3377042A4 (en) 2015-11-16 2019-05-29 The Research Institute at Nationwide Children's Hospital MATERIALS AND METHOD FOR THE TREATMENT OF TITIN-BASED MYOPATHIES AND OTHER TITINOPATHIES
MA47800A (fr) 2017-03-17 2020-01-22 Univ Newcastle Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire
AR114350A1 (es) 2018-01-31 2020-08-26 Res Institute At Nationwide Children’S Hospital Terapia genética para la distrofia muscular de cinturas del tipo 2c
BR112020025995A2 (pt) 2018-06-18 2021-03-23 Research Institute At Nationwide Children's Hospital administração de microdistrofina músculo-específica por vetor de vírus adeno-associado para tratar a distrofia muscular
WO2020006458A1 (en) 2018-06-29 2020-01-02 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
CA3131390A1 (en) 2019-02-26 2020-09-03 Research Institute Of Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
EP4488287A3 (en) 2019-08-21 2025-03-05 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
EP4087489B1 (en) 2020-01-08 2025-05-14 Vitruvia Holdings Inc. Methods and computing system for processing ultrasound image to determine health of subdermal tissue
TW202206447A (zh) 2020-04-29 2022-02-16 美商必治妥美雅史谷比公司 具血影蛋白(spectrin)融合結構域之微型化肌肉萎縮蛋白及其用途
BR112022025586A2 (pt) * 2020-06-15 2023-03-07 Res Inst Nationwide Childrens Hospital Liberação de vetor de vírus adenoassociado para distrofias musculares
RU2767335C1 (ru) * 2021-03-02 2022-03-17 Общество с ограниченной ответственностью «Марлин Биотех» Химерные белки на основе утрофина и дистрофина человека и их применение для лечения миодистрофии Дюшенна
EP4108263A3 (en) * 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
US12285497B2 (en) 2021-10-15 2025-04-29 Research Institute At Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
CN115838725B (zh) * 2022-12-30 2023-09-08 广州派真生物技术有限公司 在哺乳动物心脏中特异性启动基因的启动子序列及其应用
CN121249877A (zh) * 2025-10-17 2026-01-02 中国人民解放军总医院第一医学中心 检测杜氏肌营养不良症的生物标志物及其应用

Family Cites Families (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
ATE272123T1 (de) 1993-11-09 2004-08-15 Ohio Med College Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
AU722196B2 (en) 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
EP1983057A3 (en) 1995-09-08 2009-01-07 Genzyme Corporation Improved AAV vectors for gene therapy
US5795872A (en) 1995-09-19 1998-08-18 Pharmadigm, Inc. DNA construct for immunization
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
AU723497C (en) 1996-09-06 2001-10-11 Trustees Of The University Of Pennsylvania, The Method for recombinant adeno-associated virus-directed gene therapy
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
DK1944362T3 (en) 1997-09-05 2016-01-25 Genzyme Corp Fremgangsmåder til fremstilling af hjælpevirusfri præparater med høj titer af rekombinante AAV-vektorer
JPH11318467A (ja) 1998-05-08 1999-11-24 Japan Science & Technology Corp 短縮型ジストロフィン
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
AU2001255575B2 (en) 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
EP1287125B1 (en) 2000-04-28 2009-07-29 Asklepios Biopharmaceutical, Inc. Dna sequences encoding dystrophin minigenes and methods of use thereof
US20110097761A1 (en) 2000-07-14 2011-04-28 Chamberlain Jeffrey S Mutant muscle-specific enhancers
US6869777B2 (en) 2000-10-06 2005-03-22 Regents Of The University Of Michigan Mini-dystrophin nucleic acid sequences
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
CA2554818A1 (en) 2004-02-09 2005-08-25 Thomas Jefferson University Diagnosis and treatment of cancers with microrna located in or near cancer-associated chromosomal features
US20080044393A1 (en) 2004-07-16 2008-02-21 White Robert L Retinal dystrophin transgene and methods of use thereof
US20100184209A1 (en) 2006-02-17 2010-07-22 Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
US7892824B2 (en) 2007-01-18 2011-02-22 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma
ES2621161T3 (es) 2007-07-31 2017-07-03 The Board Of Regents Of The University Of Texas System Familia de micro-ARN que modula la fibrosis y usos de la misma
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
WO2010071454A1 (en) 2008-12-17 2010-06-24 Auckland Uniservices Limited Adeno-associated viral vectors and uses thereof
AU2012286994B2 (en) 2011-07-25 2017-08-10 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of DUX4
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
US20150045416A1 (en) 2012-01-05 2015-02-12 Hadasit Medical Research Services & Development Ltd. Methods and Compositions for Gene Delivery
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
US9624282B2 (en) 2012-11-26 2017-04-18 The Curators Of The University Of Missouri Microdystrophin peptides and methods for treating muscular dystrophy using the same
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
GB201400598D0 (en) 2014-01-14 2014-03-05 Univ Glasgow Materials and methods for modulation of tendon healing
SG11201605906UA (en) 2014-01-21 2016-08-30 Univ Bruxelles Muscle-specific nucleic acid regulatory elements and methods and use thereof
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
US9439924B2 (en) 2014-04-09 2016-09-13 University Of Houston Therapeutic miRNAs for treating heart and skeletal muscle diseases
WO2015161255A1 (en) 2014-04-18 2015-10-22 Board Of Regents Of The Nevada System Of Higher Education, On Behalf Of The University Of Nevada, Reno Methods for enhancing or decreasing the levels of mir124 and mir29 in subjects with muscular dystrophy
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
JP6832280B2 (ja) 2015-01-16 2021-02-24 ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ 新規のマイクロジストロフィンおよび使用の関連する方法
GB201507842D0 (en) 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
AU2016323575B2 (en) 2015-09-17 2022-10-27 Research Institute At Nationwide Children's Hospital Methods and materials for GALGT2 gene therapy
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
DK3442602T3 (da) 2016-04-15 2025-05-19 Res Inst Nationwide Childrens Hospital Adeno-associeret virusvektors frigørelse af mikrodystrofin til behandling af muskeldystrofi
CA2971303C (en) 2016-06-21 2026-03-03 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
BR112020025995A2 (pt) 2018-06-18 2021-03-23 Research Institute At Nationwide Children's Hospital administração de microdistrofina músculo-específica por vetor de vírus adeno-associado para tratar a distrofia muscular

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
JCI Insight. 20170504; 2(9): e93309, doi: 10.1172/jci.insight.93309
Mol Ther. 2007 Feb;15(2):320-9. doi: 10.1038/sj.mt.6300027

Also Published As

Publication number Publication date
US20230302157A1 (en) 2023-09-28
US11534501B2 (en) 2022-12-27
JP2021500352A (ja) 2021-01-07
WO2019078916A1 (en) 2019-04-25
US20210128749A1 (en) 2021-05-06
EP3697915A1 (en) 2020-08-26
WO2019078916A9 (en) 2020-07-23
EP3697915A4 (en) 2021-12-08
MA50836A (fr) 2020-08-26

Similar Documents

Publication Publication Date Title
JP7833440B2 (ja) 筋ジストロフィーを治療するためのマイクロジストロフィンのアデノ随伴ウイルスベクター送達
US20230302157A1 (en) Adeno-Associated Virus Vector Delivery of Muscle Specific Micro-Dystrophin to Treat Muscular Dystrophy
US12257317B2 (en) Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
US20220364117A1 (en) Adeno-Associated Virus Vector Delivery of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy
US12491265B2 (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
HK40118571A (zh) 腺相关病毒载体传递微肌营养不良蛋白以治疗肌营养不良症
HK40128506A (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
HK40125796A (en) Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
HK40115222A (zh) 腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症
HK40046539B (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
HK40046539A (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
EA052000B1 (ru) Доставка микродистрофина вектором на основе адено-ассоциированного вируса для лечения мышечной дистрофии
EA042315B1 (ru) Доставка микродистрофина вектором на основе аденоассоциированного вируса для лечения мышечной дистрофии

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210316

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210316

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220302

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220601

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220801

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220901

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: 20221202

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20221227

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230116

R150 Certificate of patent or registration of utility model

Ref document number: 7213238

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250