PL3596222T3 - Dostarczanie z zastosowaniem wektora wirusa związanego z adenowirusami specyficznej dla mięśni mikrodystrofiny do leczenia dystrofii mięśniowej - Google Patents
Dostarczanie z zastosowaniem wektora wirusa związanego z adenowirusami specyficznej dla mięśni mikrodystrofiny do leczenia dystrofii mięśniowejInfo
- Publication number
- PL3596222T3 PL3596222T3 PL18768395.8T PL18768395T PL3596222T3 PL 3596222 T3 PL3596222 T3 PL 3596222T3 PL 18768395 T PL18768395 T PL 18768395T PL 3596222 T3 PL3596222 T3 PL 3596222T3
- Authority
- PL
- Poland
- Prior art keywords
- microdystrophin
- adenovirus
- muscle
- delivery
- treatment
- Prior art date
Links
Classifications
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- 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
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- 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
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- 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
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- 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
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- 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
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
- A61K9/0029—Parenteral nutrition; Parenteral nutrition compositions as drug carriers
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- 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
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- 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
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- 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
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- 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
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- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
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- 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
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- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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- 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
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Immunology (AREA)
- Toxicology (AREA)
- Marine Sciences & Fisheries (AREA)
- Nutrition Science (AREA)
- Dermatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762473148P | 2017-03-17 | 2017-03-17 | |
| PCT/US2018/022881 WO2018170408A1 (en) | 2017-03-17 | 2018-03-16 | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL3596222T3 true PL3596222T3 (pl) | 2023-10-09 |
Family
ID=63523321
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL18768395.8T PL3596222T3 (pl) | 2017-03-17 | 2018-03-16 | Dostarczanie z zastosowaniem wektora wirusa związanego z adenowirusami specyficznej dla mięśni mikrodystrofiny do leczenia dystrofii mięśniowej |
Country Status (33)
Families Citing this family (33)
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| CN108463211B (zh) | 2015-11-16 | 2022-09-27 | 全国儿童医院研究所 | 用于治疗肌联蛋白类肌病和其它肌联蛋白病变的材料和方法 |
| CN110997923B (zh) * | 2017-03-17 | 2024-01-02 | 全国儿童医院研究所 | 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 |
| US11338045B2 (en) | 2017-03-17 | 2022-05-24 | Newcastle University | Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy |
| MX2020004035A (es) | 2017-10-20 | 2020-11-09 | Res Inst Nationwide Childrens Hospital | Métodos y materiales para terapia génica con nt-3. |
| AU2019216257B2 (en) | 2018-01-31 | 2025-11-20 | Research Institute At Nationwide Children's Hospital | Gene therapy for limb-girdle muscular dystrophy type 2C |
| IL324506A (en) | 2018-06-18 | 2026-01-01 | Res Inst Nationwide Childrens Hospital | Adeno-associated virus vector for delivery of muscle-specific microdystrophin for the treatment of muscular dystrophy |
| IL279789B2 (en) | 2018-06-29 | 2026-04-01 | Res Inst Nationwide Childrens Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A |
| CN113613668A (zh) | 2018-10-24 | 2021-11-05 | 百时美施贵宝公司 | 小型化肌营养不良蛋白及其用途 |
| AU2019406214A1 (en) * | 2018-12-21 | 2021-08-05 | Northwestern University | Use of annexins in preventing and treating muscle membrane injury |
| US12630840B2 (en) * | 2019-02-02 | 2026-05-19 | University of Pittsburgh—of the Commonwealth System of Higher Education | One-step gene therapy for Duchenne muscular dystrophy via gene replacement and anti-inflammation |
| KR20210133242A (ko) | 2019-02-26 | 2021-11-05 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | B-사르코글리칸의 아데노 관련 바이러스 벡터 전달 및 근이영양증의 치료 |
| EP3976806A1 (en) * | 2019-05-30 | 2022-04-06 | Solidus Biosciences, Inc. | Recombinant herpesvirales vector |
| CA3150964A1 (en) | 2019-08-21 | 2021-02-25 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy |
| CN119061198A (zh) * | 2019-08-29 | 2024-12-03 | 美国西门子医学诊断股份有限公司 | 用于检测aav脱落的试剂和方法 |
| AU2020392243A1 (en) * | 2019-11-28 | 2022-06-16 | Regenxbio Inc. | Microdystrophin gene therapy constructs and uses thereof |
| US20230040544A1 (en) * | 2019-12-16 | 2023-02-09 | Research Institute At Nationwide Children's Hospital | Compositions and methods for restoring and maintaining the dystrophin-associated protein complex (dapc) |
| US11535868B2 (en) | 2020-04-29 | 2022-12-27 | Bristol-Myers Squibb Company | Miniaturized dystrophins having spectrin fusion domains and uses thereof |
| IL300187A (en) * | 2020-08-05 | 2023-03-01 | Spacecraft Seven Llc | Gene therapy CSRP3 (protein 3 rich in cysteine and glycine) |
| WO2022029543A1 (en) * | 2020-08-06 | 2022-02-10 | Intas Pharmaceuticals Ltd. | Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy |
| KR20250099412A (ko) * | 2020-11-12 | 2025-07-01 | 프리시젼 바이오사이언시스 인코포레이티드 | 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제 |
| EP4086276A1 (en) | 2021-05-03 | 2022-11-09 | Université d'Aix-Marseille | Composition for treating dysferlinopathy |
| BR112023024078A2 (pt) * | 2021-05-17 | 2024-01-30 | Sarepta Therapeutics Inc | Produção de vetores de aav recombinantes para o tratamento de distrofia muscular |
| 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 |
| WO2023015304A1 (en) * | 2021-08-05 | 2023-02-09 | Insmed Incorporated | Adeno-associated virus particles and methods of use thereof |
| JP2024531138A (ja) * | 2021-08-11 | 2024-08-29 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | 筋ジストロフィーを処置するための組成物および方法 |
| 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 |
| CN114316070B (zh) * | 2021-12-29 | 2022-11-15 | 上海勉亦生物科技有限公司 | 用于治疗肌营养不良症的转基因表达盒 |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| CN118460618A (zh) * | 2022-04-19 | 2024-08-09 | 康霖生物科技（杭州）有限公司 | 一种用于遗传性凝血因子缺乏病治疗的核酸构建体 |
| WO2023248251A1 (en) * | 2022-06-24 | 2023-12-28 | Indian Institute Of Technology Kanpur | An optimized aav vector for gene therapy of muscular dystrophy |
| CN115819546B (zh) * | 2022-08-19 | 2025-12-05 | 成都金唯科生物科技有限公司 | 表达微缩抗肌营养不良蛋白基因的腺相关病毒载体及其应用 |
| WO2026076414A1 (en) * | 2024-10-03 | 2026-04-09 | General Medicines Llc | Compositions and methods for modulating expression of therapeutic transgenes |
| WO2026080823A1 (en) | 2024-10-10 | 2026-04-16 | Research Institute At Nationwide Children's Hospital | Methods for diagnosing and treating muscular dystrophy diseases |
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| WO1996017947A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
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| AU2015208247B2 (en) * | 2014-01-21 | 2021-05-27 | Universiteit Gent | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
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| GB201507842D0 (en) * | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
| WO2017181011A1 (en) * | 2016-04-15 | 2017-10-19 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of microrna-29 to treat muscular dystrophy |
| CN110997923B (zh) * | 2017-03-17 | 2024-01-02 | 全国儿童医院研究所 | 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 |
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