TWI856016B - 腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症 - Google Patents

腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症 Download PDF

Info

Publication number
TWI856016B
TWI856016B TW108121156A TW108121156A TWI856016B TW I856016 B TWI856016 B TW I856016B TW 108121156 A TW108121156 A TW 108121156A TW 108121156 A TW108121156 A TW 108121156A TW I856016 B TWI856016 B TW I856016B
Authority
TW
Taiwan
Prior art keywords
raav
seq
administration
nucleotides
mhck7
Prior art date
Application number
TW108121156A
Other languages
English (en)
Chinese (zh)
Other versions
TW202000915A (zh
Inventor
克拉帕克 路易斯 羅帝諾
傑瑞 R 麥德爾
Original Assignee
美國全美兒童醫院之研究學會
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 美國全美兒童醫院之研究學會 filed Critical 美國全美兒童醫院之研究學會
Publication of TW202000915A publication Critical patent/TW202000915A/zh
Application granted granted Critical
Publication of TWI856016B publication Critical patent/TWI856016B/zh

Links

Classifications

    • 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
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1719—Muscle proteins, e.g. myosin or actin
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • 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
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
TW108121156A 2018-06-18 2019-06-18 腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症 TWI856016B (zh)

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US201862686668P 2018-06-18 2018-06-18
US62/686,668 2018-06-18
US201862740402P 2018-10-02 2018-10-02
US62/740,402 2018-10-02
US201862752841P 2018-10-30 2018-10-30
US62/752,841 2018-10-30
US201962823649P 2019-03-25 2019-03-25
US62/823,649 2019-03-25
US201962860220P 2019-06-11 2019-06-11
US62/860,220 2019-06-11
WOPCT/US2019/037489 2019-06-17
PCT/US2019/037489 WO2019245973A1 (en) 2018-06-18 2019-06-17 Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy

Publications (2)

Publication Number Publication Date
TW202000915A TW202000915A (zh) 2020-01-01
TWI856016B true TWI856016B (zh) 2024-09-21

Family

ID=67544316

Family Applications (1)

Application Number Title Priority Date Filing Date
TW108121156A TWI856016B (zh) 2018-06-18 2019-06-18 腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症

Country Status (28)

Country Link
US (1) US12491265B2 (https=)
EP (2) EP3807413B1 (https=)
JP (3) JP7646362B2 (https=)
KR (2) KR20210022038A (https=)
CN (2) CN120393053A (https=)
AU (1) AU2019290544B2 (https=)
BR (1) BR112020025995A2 (https=)
CA (1) CA3103485A1 (https=)
CL (1) CL2020003289A1 (https=)
CO (1) CO2021000227A2 (https=)
DK (1) DK3807413T3 (https=)
ES (1) ES3038120T3 (https=)
FI (1) FI3807413T3 (https=)
HR (1) HRP20250932T1 (https=)
HU (1) HUE072475T2 (https=)
IL (2) IL279521B2 (https=)
LT (1) LT3807413T (https=)
MX (3) MX2020013888A (https=)
MY (1) MY208145A (https=)
PL (1) PL3807413T3 (https=)
PT (1) PT3807413T (https=)
RS (1) RS67118B1 (https=)
SA (1) SA520420833B1 (https=)
SG (1) SG11202012450QA (https=)
SI (1) SI3807413T1 (https=)
SM (1) SMT202500303T1 (https=)
TW (1) TWI856016B (https=)
WO (1) WO2019245973A1 (https=)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102877920B1 (ko) 2015-11-16 2025-10-30 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법
ES2977730T3 (es) 2016-04-15 2024-08-29 Res Inst Nationwide Childrens Hospital Administración de microARN-29 y micro-distrofina por virus adenoasociado para tratar la distrofia muscular
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
MA50836A (fr) 2017-10-18 2020-08-26 Res Inst Nationwide Childrens Hospital Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles 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
MY208145A (en) 2018-06-18 2025-04-18 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
KR20210028162A (ko) 2018-06-29 2021-03-11 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
AU2020229340A1 (en) 2019-02-26 2021-09-16 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy
RS65421B1 (sr) 2019-08-21 2024-05-31 Res Inst Nationwide Childrens Hospital Isporuka adeno-asociranog virusnog vektora alfa-sarkoglikana i lečenje mišićne distrofije
WO2021138286A1 (en) * 2020-01-03 2021-07-08 The Board Of Regents Of The University Of Texas System Self-complementary aav delivery system for crispr/cas9
IL297753A (en) 2020-04-29 2022-12-01 Bristol Myers Squibb Co Minimized dystrophins with spectrin fusion domains and uses thereof
IL299094A (en) * 2020-06-15 2023-02-01 Res Inst Nationwide Childrens Hospital Administration of an adeno-associated virus vector for muscular dystrophies
US20240003898A1 (en) 2020-10-30 2024-01-04 Pfizer Inc. Methods for measuring dystrophin in tissue samples
AU2022279062A1 (en) 2021-05-17 2024-01-04 Sarepta Therapeutics, Inc. Production of recombinant aav vectors for treating muscular dystrophy
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
US12417980B2 (en) 2022-05-24 2025-09-16 Taiwan Semiconductor Manufacturing Company, Ltd. First metal structure, layout, and method
CN115029360B (zh) * 2022-05-30 2024-08-02 上海勉亦生物科技有限公司 用于治疗粘多糖贮积症iiia型的转基因表达盒
EP4590838A1 (en) 2022-09-23 2025-07-30 Sarepta Therapeutics, Inc. Recombinant aav vectors for treating muscular dystrophy
WO2026080823A1 (en) 2024-10-10 2026-04-16 Research Institute At Nationwide Children's Hospital Methods for diagnosing and treating muscular dystrophy diseases

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017181015A1 (en) * 2016-04-15 2017-10-19 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy

Family Cites Families (117)

* 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
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
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
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation 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
JP2001500497A (ja) 1996-09-06 2001-01-16 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法
US5965542A (en) 1997-03-18 1999-10-12 Inex Pharmaceuticals Corp. Use of temperature to control the size of cationic liposome/plasmid DNA complexes
JP4119607B2 (ja) 1997-08-15 2008-07-16 チッソ株式会社 多分散性プロピレン重合体およびその製造方法
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6410328B1 (en) 1998-02-03 2002-06-25 Protiva Biotherapeutics Inc. Sensitizing cells to compounds using lipid-mediated gene and compound delivery
CA2321837C (en) 1998-02-03 2011-08-16 Inex Pharmaceuticals Corporation Systemic delivery of serum stable plasmid lipid particles for cancer therapy
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
US6211140B1 (en) 1999-07-26 2001-04-03 The Procter & Gamble Company Cationic charge boosting systems
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
ES2330615T3 (es) 2000-04-28 2009-12-14 Asklepios Biopharmaceutical, Inc. Secuencias de adn que codifican para minigenes de distrofina y metodos de uso de las mismas.
ATE373095T1 (de) 2000-07-14 2007-09-15 Univ Michigan Mutierte muskelspezifische enhancer
EP1366160B1 (en) 2000-10-06 2008-07-09 The Regents Of The University Of Michigan Mini-dystrophin nucleic acid and peptide sequences
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US20060194265A1 (en) 2001-10-23 2006-08-31 Morris David W Novel therapeutic targets in cancer
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
CA2532228C (en) 2003-07-16 2017-02-14 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
US6927663B2 (en) 2003-07-23 2005-08-09 Cardiac Pacemakers, Inc. Flyback transformer wire attach method to printed circuit board
EP1664316B1 (en) 2003-09-15 2012-08-29 Protiva Biotherapeutics Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
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
US20070053845A1 (en) 2004-03-02 2007-03-08 Shiladitya Sengupta Nanocell drug delivery system
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
US20080044393A1 (en) 2004-07-16 2008-02-21 White Robert L Retinal dystrophin transgene and methods of use thereof
CN101267805A (zh) 2005-07-27 2008-09-17 普洛体维生物治疗公司 制造脂质体的系统和方法
WO2007095387A2 (en) 2006-02-17 2007-08-23 Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
US9149564B2 (en) 2006-06-23 2015-10-06 The Regents Of The University Of California Articles comprising large-surface-area bio-compatible materials and methods for making and using them
CN101711164B (zh) 2007-01-18 2014-06-04 密苏里-哥伦比亚大学 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因
EP2182969B1 (en) 2007-07-31 2016-11-16 The Board of Regents of The University of Texas System A micro-rna family that modulates fibrosis and uses thereof
ES2639852T3 (es) 2007-10-26 2017-10-30 Academisch Ziekenhuis Leiden Medios y métodos para contrarrestar los trastornos musculares
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
CN111808084A (zh) 2008-11-10 2020-10-23 阿布特斯生物制药公司 用于递送治疗剂的新型脂质和组合物
WO2010071454A1 (en) 2008-12-17 2010-06-24 Auckland Uniservices Limited Adeno-associated viral vectors and uses thereof
EP2421971B1 (en) 2009-04-24 2016-07-06 BioMarin Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011000107A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
WO2011004395A1 (en) 2009-07-06 2011-01-13 Intas Biopharmaceuticals Limited Microemulsion formulation for biologicals
WO2011066651A1 (en) 2009-12-01 2011-06-09 Protiva Biotherapeutics, Inc. Snalp formulations containing antioxidants
EP3296398A1 (en) 2009-12-07 2018-03-21 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
EP3494963A1 (en) 2009-12-18 2019-06-12 The University of British Columbia Methods and compositions for delivery of nucleic acids
CA2800497C (en) 2010-05-10 2019-03-12 The Regents Of The University Of California Methods for preparing ratiometric combinatorial nanoparticules
US20130123338A1 (en) 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
KR20190039347A (ko) 2010-06-03 2019-04-10 알닐람 파마슈티칼스 인코포레이티드 활성제의 전달을 위한 생분해성 지질
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
US8691750B2 (en) 2011-05-17 2014-04-08 Axolabs Gmbh Lipids and compositions for intracellular delivery of biologically active compounds
WO2013016126A1 (en) 2011-07-22 2013-01-31 The General Hospital Corporation Therapeutic nanoparticles and methods of use thereof
WO2013016058A1 (en) 2011-07-22 2013-01-31 Merck Sharp & Dohme Corp. Novel bis-nitrogen containing cationic lipids for oligonucleotide delivery
EP2736539B1 (en) 2011-07-25 2017-08-23 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
EP3456317B1 (en) 2011-09-27 2025-09-24 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
US8762704B2 (en) 2011-09-29 2014-06-24 Apple Inc. Customized content for electronic devices
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
AU2012347605B2 (en) 2011-12-07 2017-09-21 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
WO2013086354A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
WO2013086373A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
WO2013102904A1 (en) 2012-01-05 2013-07-11 Hadasit Medical Research Services & Development Ltd. Methods and compositions for gene delivery
MX367100B (es) 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
WO2014008334A1 (en) 2012-07-06 2014-01-09 Alnylam Pharmaceuticals, Inc. Stable non-aggregating nucleic acid lipid particle formulations
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
MX388221B (es) 2013-04-20 2025-03-19 Res Institute At Nationwide Children´S Hospital ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2.
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
PL3097197T3 (pl) 2014-01-21 2021-06-28 Vrije Universiteit Brussel Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie
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
WO2015157306A1 (en) 2014-04-09 2015-10-15 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
HUE060907T2 (hu) 2014-06-25 2023-04-28 Acuitas Therapeutics Inc Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
CA2957661A1 (en) 2014-08-09 2016-02-18 Kevin FLANIGAN Methods and materials for activating an internal ribosome entry site in exon 5 of the dmd gene
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
PT3313829T (pt) 2015-06-29 2024-07-08 Acuitas Therapeutics Inc Formulações de lípidos e de nanopartículas lipídicas para a administração de ácidos nucleicos
IL258005B2 (en) 2015-09-17 2025-01-01 Res Inst Nationwide Childrens Hospital Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2
HRP20230209T1 (hr) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
ES3034857T3 (en) 2015-11-12 2025-08-25 The Res Institute At Nationwide Childrens Hospital Methods of treating muscular dystrophy
WO2017117528A1 (en) 2015-12-30 2017-07-06 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
LT3442600T (lt) * 2016-04-15 2024-05-27 Research Institute At Nationwide Children's Hospital Su adeno virusu susijusio viruso vektoriaus iš β-sarkoglikano ir mikro- rnr-29 pristatymas ir raumenų distrofijos gydymas
CA2971303C (en) 2016-06-21 2026-03-03 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
JP7676111B2 (ja) * 2017-03-17 2025-05-14 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送達
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
CN107369008A (zh) 2017-07-17 2017-11-21 北京京东金融科技控股有限公司 用于提高票据交易安全性的管理方法、装置及系统
US20200164008A1 (en) 2017-07-17 2020-05-28 Spark Therapeutics, Inc. Apheresis methods and uses
MA50836A (fr) 2017-10-18 2020-08-26 Res Inst Nationwide Childrens Hospital Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles pour traiter la dystrophie musculaire
WO2019089828A1 (en) 2017-10-31 2019-05-09 Acuitas Therapeutics, Inc. Lamellar lipid nanoparticles
WO2019118806A1 (en) 2017-12-14 2019-06-20 Solid Biosciences Inc. Non-viral production and delivery of genes
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
WO2019177550A1 (en) 2018-03-10 2019-09-19 Koc Universitesi Therapeutic nanoparticles containing argonaute for microrna delivery and compositions and methods using same
US20210139550A1 (en) 2018-04-03 2021-05-13 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
MY208145A (en) 2018-06-18 2025-04-18 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
KR20210028162A (ko) 2018-06-29 2021-03-11 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
CA3116576A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
WO2020123645A1 (en) 2018-12-12 2020-06-18 Solid Biosciences Inc. Combination therapy for treating muscular dystrophy
AU2020229340A1 (en) 2019-02-26 2021-09-16 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of B-sarcoglycan and the treatment of muscular dystrophy
RS65421B1 (sr) 2019-08-21 2024-05-31 Res Inst Nationwide Childrens Hospital Isporuka adeno-asociranog virusnog vektora alfa-sarkoglikana i lečenje mišićne distrofije
US12391745B2 (en) 2020-06-15 2025-08-19 Sarepta Therapeutics, Inc. Adeno-associated virus antibodies and fragments thereof

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017181015A1 (en) * 2016-04-15 2017-10-19 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
期刊 Dongsheng Duan, "Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy", Mol Ther, Review, 2018 Oct 3; 26(10):2337-2356. Epub 2018 Jul 17.
期刊 Maja Z Salva et al , "Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle", Mol Ther. 2007 Feb;15(2): 320-9.;期刊 Dongsheng Duan, "Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy", Mol Ther, Review, 2018 Oct 3; 26(10):2337-2356. Epub 2018 Jul 17. *

Also Published As

Publication number Publication date
JP2021527657A (ja) 2021-10-14
AU2019290544B2 (en) 2026-01-22
JP2025148438A (ja) 2025-10-07
KR20250092292A (ko) 2025-06-23
BR112020025995A2 (pt) 2021-03-23
EP4591888A2 (en) 2025-07-30
IL279521B1 (en) 2025-12-01
PL3807413T3 (pl) 2025-11-03
TW202000915A (zh) 2020-01-01
ES3038120T3 (en) 2025-10-09
CL2020003289A1 (es) 2021-07-09
MY208145A (en) 2025-04-18
LT3807413T (lt) 2025-09-10
EP3807413B1 (en) 2025-05-21
AU2019290544A1 (en) 2021-01-28
SMT202500303T1 (it) 2025-09-12
SA520420833B1 (ar) 2025-05-22
MX2024015879A (es) 2025-02-10
HUE072475T2 (hu) 2025-11-28
EP4591888A3 (en) 2026-01-21
JP7853354B2 (ja) 2026-04-28
CN112823210A (zh) 2021-05-18
IL279521B2 (en) 2026-04-01
TW202525348A (zh) 2025-07-01
MX2020013888A (es) 2021-05-27
RS67118B1 (sr) 2025-09-30
DK3807413T3 (da) 2025-08-25
CN120393053A (zh) 2025-08-01
JP2024096248A (ja) 2024-07-12
PT3807413T (pt) 2025-08-19
EP3807413A1 (en) 2021-04-21
IL279521A (en) 2021-01-31
KR20210022038A (ko) 2021-03-02
CO2021000227A2 (es) 2021-01-18
US20210260218A1 (en) 2021-08-26
CA3103485A1 (en) 2019-12-26
JP7646362B2 (ja) 2025-03-17
CN112823210B (zh) 2025-04-18
HRP20250932T1 (hr) 2025-09-26
FI3807413T3 (fi) 2025-08-13
SI3807413T1 (sl) 2025-12-31
WO2019245973A1 (en) 2019-12-26
SG11202012450QA (en) 2021-01-28
IL324506A (en) 2026-01-01
US12491265B2 (en) 2025-12-09
MX2025006507A (es) 2025-07-01

Similar Documents

Publication Publication Date Title
TWI856016B (zh) 腺相關病毒載體遞送肌肉特異性微小肌縮蛋白以治療肌肉萎縮症
JP7752157B2 (ja) 肢帯型筋ジストロフィー２ｃ型のための遺伝子治療
US20220364117A1 (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
JP7213238B2 (ja) 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
JP2026022658A (ja) 筋ジストロフィーのためのアデノ随伴ウイルスベクター送達
AU2022279062A1 (en) Production of recombinant aav vectors for treating muscular dystrophy
HK40126715A (zh) 用於治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送
HK40128506A (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
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
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