[go: up one dir, main page]

SI4017871T1 - Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije - Google Patents

Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije

Info

Publication number
SI4017871T1
SI4017871T1 SI202030411T SI202030411T SI4017871T1 SI 4017871 T1 SI4017871 T1 SI 4017871T1 SI 202030411 T SI202030411 T SI 202030411T SI 202030411 T SI202030411 T SI 202030411T SI 4017871 T1 SI4017871 T1 SI 4017871T1
Authority
SI
Slovenia
Prior art keywords
sarcoglycan
adeno
alpha
treatment
associated virus
Prior art date
Application number
SI202030411T
Other languages
English (en)
Inventor
Louise Rodino-Klapac
Danielle Griffin
Jerry R. Mendell
Original Assignee
Research Institute At Nationwide Children's Hospital
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=72340432&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=SI4017871(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Research Institute At Nationwide Children's Hospital filed Critical Research Institute At Nationwide Children's Hospital
Publication of SI4017871T1 publication Critical patent/SI4017871T1/sl

Links

Classifications

    • 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
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
SI202030411T 2019-08-21 2020-08-21 Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije SI4017871T1 (sl)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962889749P 2019-08-21 2019-08-21
US202063014934P 2020-04-24 2020-04-24
US202063022843P 2020-05-11 2020-05-11
EP20765424.5A EP4017871B1 (en) 2019-08-21 2020-08-21 Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
PCT/US2020/047339 WO2021035120A1 (en) 2019-08-21 2020-08-21 Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy

Publications (1)

Publication Number Publication Date
SI4017871T1 true SI4017871T1 (sl) 2024-06-28

Family

ID=72340432

Family Applications (1)

Application Number Title Priority Date Filing Date
SI202030411T SI4017871T1 (sl) 2019-08-21 2020-08-21 Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije

Country Status (25)

Country Link
US (2) US12258573B2 (sl)
EP (3) EP4488287A3 (sl)
JP (2) JP7532503B2 (sl)
KR (1) KR20220092489A (sl)
CN (2) CN120400170A (sl)
AU (1) AU2020331987B2 (sl)
BR (1) BR112022003142A2 (sl)
CA (1) CA3150964A1 (sl)
CO (1) CO2022002864A2 (sl)
DK (1) DK4017871T5 (sl)
ES (1) ES2978666T3 (sl)
FI (1) FI4017871T3 (sl)
HR (1) HRP20240505T1 (sl)
HU (1) HUE066215T2 (sl)
IL (2) IL290734A (sl)
LT (1) LT4017871T (sl)
MX (1) MX2022002132A (sl)
MY (1) MY210230A (sl)
PL (1) PL4017871T3 (sl)
PT (1) PT4017871T (sl)
RS (1) RS65421B1 (sl)
SI (1) SI4017871T1 (sl)
SM (1) SMT202400152T1 (sl)
TW (1) TWI873172B (sl)
WO (1) WO2021035120A1 (sl)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL259441B2 (en) 2015-11-16 2024-01-01 Res Inst Nationwide Childrens Hospital Materials and methods for treatment of titin-based myopathies and other titinopaties
EP3596112A2 (en) 2017-03-17 2020-01-22 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
CN119770682A (zh) 2018-01-31 2025-04-08 国家儿童医院研究所 2c型肢带型肌营养不良的基因疗法
JP7646362B2 (ja) 2018-06-18 2025-03-17 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 筋ジストロフィーを治療するための筋肉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デリバリー
MX2020014119A (es) 2018-06-29 2021-06-18 Res Inst Nationwide Childrens Hospital Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a.
SG11202109113TA (en) 2019-02-26 2021-09-29 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
SI4017871T1 (sl) * 2019-08-21 2024-06-28 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije
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
EP4198046A1 (en) 2021-12-16 2023-06-21 Genethon Alpha-sarcoglycan gene transfer increase using modified itr sequences
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders

Family Cites Families (110)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0127839B1 (en) 1983-05-27 1992-07-15 THE TEXAS A&M UNIVERSITY SYSTEM Method for producing a recombinant baculovirus expression vector
DK518384A (da) 1984-01-31 1985-07-01 Idaho Res Found Vektor til fremstilling af et gen-produkt i insektceller, fremgangsmaade til dens fremstilling samt dens anvendelse
US5449616A (en) 1990-05-23 1995-09-12 University Of Iowa Research Foundation Nucleic acid encoding dystrophin-associated protein
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
WO1995003392A1 (en) 1993-07-23 1995-02-02 Unichema Chemie Bv Process for producing transparent soap material
JPH09509564A (ja) 1993-11-09 1997-09-30 ターゲテッド ジェネティックス コーポレイション 高力価組換えａａｖベクターの生成
WO1995013392A1 (en) 1993-11-09 1995-05-18 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
WO1996017947A1 (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
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびａａｖの精製
CA2230758A1 (en) 1995-09-08 1997-03-13 Genzyme Corporation Improved aav vectors for gene therapy
US5672694A (en) 1995-10-24 1997-09-30 University Of Iowa Research Foundation β-sarcoglycan nucleic acid sequence, and nucleic acid probes
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
ATE465267T1 (de) 1996-09-06 2010-05-15 Univ Pennsylvania Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen
US5843078A (en) 1997-07-01 1998-12-01 Sharkey; Hugh R. Radio frequency device for resurfacing skin and method
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ES2399232T3 (es) 1997-09-05 2013-03-26 Genzyme Corporation Procedimientos para la generación de preparaciones de vectores de AAV recombinantes de títulos altos exentas de auxiliares
WO1999043360A1 (en) 1998-02-26 1999-09-02 The Trustees Of The University Of Pennsylvania Stable protection from dystrophic sarcolemmal degeneration and restoration of the sarcoglycan complex
US6262035B1 (en) 1998-10-01 2001-07-17 University Of Iowa Research Foundation Gene replacement therapy for muscular dystrophy
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
US6632800B1 (en) 1999-08-17 2003-10-14 Mayo Foundation For Medical Education And Research System for monitoring the expression of transgenes
JP4520569B2 (ja) 2000-02-18 2010-08-04 照彦 豊岡 拡張型心筋症の遺伝子治療剤
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US20040126762A1 (en) 2002-12-17 2004-07-01 Morris David W. Novel compositions and methods in cancer
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
WO2003074714A1 (en) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Baculovirus expression system
AU2003223775A1 (en) 2002-04-30 2003-11-17 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
US20030225260A1 (en) 2002-04-30 2003-12-04 Snyder Richard O. Production of recombinant AAV virions
DK1751275T3 (da) 2004-06-01 2017-11-27 Avigen Inc Sammensætninger og fremgangsmåder til forebyggelse af aggregering af aav-vektorer
US7972593B2 (en) 2004-06-10 2011-07-05 Saint Louis University Delivery of therapeutic agents to the bone
WO2006039218A2 (en) 2004-09-30 2006-04-13 The Trustees Of The University Of Pennsylvania Perfusion circuit and use therein in targeted delivery of macromolecules
JP2006121961A (ja) 2004-10-28 2006-05-18 Univ Of Tokushima 顎骨骨幹異形成症ｇｄｄの原因遺伝子ｇｄｄ１とその用途
US7883858B2 (en) 2005-01-27 2011-02-08 Institute For Systems Biology Methods for identifying and monitoring drug side effects
US7788045B2 (en) 2005-09-01 2010-08-31 Meditasks, Llc Systems and method for homeostatic blood states
EP1938104A2 (en) 2005-10-17 2008-07-02 Institute for Systems Biology Tissue-and serum-derived glycoproteins and methods of their use
WO2007049095A1 (en) 2005-10-25 2007-05-03 Cellectis Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof
US9198984B2 (en) 2006-04-28 2015-12-01 The Trustees Of The University Of Pennsylvania Scalable production method for AAV
WO2008016356A2 (en) 2006-08-02 2008-02-07 Genizon Biosciences Genemap of the human genes associated with psoriasis
US8586006B2 (en) 2006-08-09 2013-11-19 Institute For Systems Biology Organ-specific proteins and methods of their use
US20100093089A1 (en) 2006-11-09 2010-04-15 Eduardo Marban Dedifferentiation of adult mammalian cardiomyocytes into cardiac stem cells
WO2008088895A2 (en) 2007-01-18 2008-07-24 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma
FR2919305B1 (fr) 2007-07-26 2009-09-18 Genethon Ass Loi De 1901 Vecteurs viraux adeno-associes pour l'expression de la dysferline.
GB0715087D0 (en) 2007-08-03 2007-09-12 Summit Corp Plc Drug combinations for the treatment of duchenne muscular dystrophy
ES2639852T3 (es) 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
CN102027123B (zh) 2008-03-14 2016-03-16 人体酶有限公司 使用人细胞表达系统重组产生可靠的人蛋白质
US20090280103A1 (en) 2008-04-04 2009-11-12 Martin Flueck Regulation of muscle repair
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
US20100026655A1 (en) 2008-07-31 2010-02-04 Avago Technologies Ecbu Ip (Singapore) Pte. Ltd. Capacitive Touchscreen or Touchpad for Finger or Stylus
US8729041B2 (en) 2008-12-03 2014-05-20 The Johns Hopkins University Compositions and methods for treating hepatic neoplasia
US20110023139A1 (en) 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in cardiovascular disease
US20100247495A1 (en) 2009-03-30 2010-09-30 Tom Ichim Treatment of Muscular Dystrophy
US9653655B2 (en) 2009-04-15 2017-05-16 The Regents Of The University Of California Light emitting diodes with zinc oxide current spreading and light extraction layers deposited from low temperature aqueous solution
US8946451B2 (en) 2009-10-05 2015-02-03 Catabasis Pharmaceuticals, Inc. Lipoic acid acylated salicylate derivatives and their uses
FR2962041B1 (fr) 2010-07-01 2012-07-27 Genethon Inhibiteurs de la calpaine 3 pour le traitement de dystrophies musculaires et de cardiomyopathies
EP4488370A2 (en) 2011-07-25 2025-01-08 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expresssion of dux4
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
SG11201404956PA (en) 2012-02-17 2014-09-26 Philadelphia Children Hospital Aav vector compositions and methods for gene transfer to cells, organs and tissues
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
HK1206601A1 (en) 2012-04-02 2016-01-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
RS59199B1 (sr) 2012-05-25 2019-10-31 Univ California Metode i jedinjenja za rnk-upravljanu ciljanu dnk modifikaciju i za rnk- upravljanu modulaciju transkripta
LT3421602T (lt) 2012-09-06 2021-05-25 The University Of Chicago Priešprasmiai polinukleotidai, skirti indukuoti egzono praleidimą ir distrofijų gydymo būdai
US20150247196A1 (en) 2012-09-07 2015-09-03 INSERM (Institut National de la Santé et de la Recherche Médicale) In vitro genetic diagnostic of inherited neuromuscular disorders
MX369519B (es) 2012-09-25 2019-11-11 Genzyme Corp Oligonucleotidos antisentido morfolino unidos a peptido para el tratamiento de distrofia miotonica.
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
EP3825401A1 (en) 2012-12-12 2021-05-26 The Broad Institute, Inc. Crispr-cas component systems, methods and compositions for sequence manipulation
RU2721275C2 (ru) 2012-12-12 2020-05-18 Те Брод Инститьют, Инк. Доставка, конструирование и оптимизация систем, способов и композиций для манипуляции с последовательностями и применения в терапии
CN113528577B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 用于序列操纵的系统、方法和优化的指导组合物的工程化
FR3004463A1 (fr) 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective
KR20160034901A (ko) 2013-06-17 2016-03-30 더 브로드 인스티튜트, 인코퍼레이티드 서열 조작에 최적화된 ｃｒｉｓｐｒ-ｃａｓ 이중 닉카아제 시스템, 방법 및 조성물
ITTO20130669A1 (it) 2013-08-05 2015-02-06 Consiglio Nazionale Ricerche Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
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
HUE037179T2 (hu) 2013-10-08 2018-08-28 Ystem S R L Gyógyászati kompozíciók izom-rendellenességek kezelésére
US9850497B2 (en) 2013-11-04 2017-12-26 Regents Of The University Of Minnesota Gene targeting methods and tools
BR112016013213A2 (pt) 2013-12-12 2017-12-05 Massachusetts Inst Technology administração, uso e aplicações terapêuticas dos sistemas crispr-cas e composições para visar distúrbios e doenças usando componentes de administração de partículas
PL3097197T3 (pl) 2014-01-21 2021-06-28 Vrije Universiteit Brussel Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie
JP6202701B2 (ja) 2014-03-21 2017-09-27 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プログラム
DE102014207498A1 (de) 2014-04-17 2015-10-22 Universitätsklinikum Hamburg-Eppendorf Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark
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
IL315273A (en) 2015-01-16 2024-10-01 Univ Washington Novel micro-dystrophins and related methods of use
IL259441B2 (en) 2015-11-16 2024-01-01 Res Inst Nationwide Childrens Hospital Materials and methods for treatment of titin-based myopathies and other titinopaties
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
MA45477A (fr) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
JP7079733B2 (ja) * 2016-04-15 2022-06-02 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 β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療
WO2017180857A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia a
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
SI3596222T1 (sl) 2017-03-17 2024-05-31 Research Institute At Nationwide Children's Hospital Dostava mišično specifičnega mikro-distrofina, s pomočjo adeno povezanega virusnega vektorja, za zdravljenje mišične distrofije
EP3596112A2 (en) 2017-03-17 2020-01-22 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
JP7213238B2 (ja) 2017-10-18 2023-01-26 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 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
JP2021500029A (ja) 2017-10-20 2021-01-07 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 Ｎｔ−３遺伝子治療のための方法及び材料
TW201936201A (zh) 2017-12-14 2019-09-16 美商堅固生物科技公司 基因之非病毒生產及遞送
CN119770682A (zh) 2018-01-31 2025-04-08 国家儿童医院研究所 2c型肢带型肌营养不良的基因疗法
WO2019195362A1 (en) 2018-04-03 2019-10-10 Curators Of The University Of Missouri Hinges 1 and/or 4 modified dystrophins for dystrophinopathy therapy
US12152242B2 (en) 2018-04-23 2024-11-26 The Curators Of The University Of Missouri CRISPR therapy
JP7646362B2 (ja) 2018-06-18 2025-03-17 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 筋ジストロフィーを治療するための筋肉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デリバリー
MX2020014119A (es) 2018-06-29 2021-06-18 Res Inst Nationwide Childrens Hospital Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a.
CN113646004A (zh) 2018-12-12 2021-11-12 坚固生物科技公司 用于治疗肌营养不良的组合疗法
SG11202109113TA (en) 2019-02-26 2021-09-29 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
SI4017871T1 (sl) * 2019-08-21 2024-06-28 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije
US20210393801A1 (en) 2020-06-15 2021-12-23 Research Institute At Nationwide Children's Hospital Adeno-Associated Virus Vector Delivery for Muscular Dystrophies
MX2022016243A (es) 2020-06-17 2023-03-08 Acceleron Pharma Inc Antagonistas de actrii-alk4 y métodos para el tratamiento contra insuficiencia cardíaca.
US20230390417A1 (en) 2022-06-02 2023-12-07 Research Institute At Nationwide Children's Hospital Recombinant Adeno-Associated Virus Products and Methods for Treating Limb Girdle Muscular Dystrophy 2A

Also Published As

Publication number Publication date
TWI873172B (zh) 2025-02-21
MY210230A (en) 2025-09-04
AU2020331987B2 (en) 2025-10-16
KR20220092489A (ko) 2022-07-01
HRP20240505T1 (hr) 2024-07-05
PL4017871T3 (pl) 2024-07-08
JP2022544627A (ja) 2022-10-19
CA3150964A1 (en) 2021-02-25
US12258573B2 (en) 2025-03-25
CN114945583A (zh) 2022-08-26
IL290734A (en) 2022-04-01
EP4488287A2 (en) 2025-01-08
DK4017871T5 (da) 2024-08-05
EP4374880A2 (en) 2024-05-29
AU2020331987A1 (en) 2022-03-31
US20220290180A1 (en) 2022-09-15
PT4017871T (pt) 2024-04-22
CN120400170A (zh) 2025-08-01
TW202120532A (zh) 2021-06-01
FI4017871T3 (fi) 2024-04-22
JP7532503B2 (ja) 2024-08-13
ES2978666T3 (es) 2024-09-17
WO2021035120A1 (en) 2021-02-25
IL323311A (en) 2025-11-01
BR112022003142A2 (pt) 2022-05-17
RS65421B1 (sr) 2024-05-31
MX2022002132A (es) 2022-05-18
EP4017871A1 (en) 2022-06-29
EP4017871B1 (en) 2024-01-17
DK4017871T3 (da) 2024-04-22
US20250122531A1 (en) 2025-04-17
CN114945583B (zh) 2025-04-29
HUE066215T2 (hu) 2024-07-28
EP4488287A3 (en) 2025-03-05
EP4374880A3 (en) 2024-08-14
JP2024156816A (ja) 2024-11-06
SMT202400152T1 (it) 2024-05-14
LT4017871T (lt) 2024-05-10
CO2022002864A2 (es) 2022-07-08

Similar Documents

Publication Publication Date Title
IL285849A (en) Delivery of b-sarcoglycan using an adeno-associated virus vector and treatment of muscular dystrophy
PT4017871T (pt) Distribuição de vetor de vírus adeno-associado de alfasarcoglicano e o tratamento da distrofia muscular
ZA201806863B (en) Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy
ZA201906608B (en) Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
IL279521A (en) Adeno-associated virus vector for administration of muscle-specific microdystrophin for the treatment of muscular dystrophy
IL292814A (en) Improved vectors of adeno-associated virus, and their delivery to the central nervous system
IL279789A (en) Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
IL287214A (en) Adeno-associated virus vector formulations and methods
DK3596222T3 (da) Adenoassocieret virusvektorfremføring af muskelspecifik mikro-dystrophin til behandling af muskeldystrofi
EP3697915A4 (en) ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION SPECIFIC TO MUSCLES TO TREAT MUSCLE DYSTROPHY
HK40065393A (en) Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
IL291704A (en) Systemic administration of peptides to treat spinal cord injury and/or remyelination
HK40049735A (en) Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type iva
HK40052077A (en) Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
HK40035525A (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy