ZA201803956B - Adeno-associated viral vectors useful in treatment of spinal muscular atropy - Google Patents
Adeno-associated viral vectors useful in treatment of spinal muscular atropyInfo
- Publication number
- ZA201803956B ZA201803956B ZA2018/03956A ZA201803956A ZA201803956B ZA 201803956 B ZA201803956 B ZA 201803956B ZA 2018/03956 A ZA2018/03956 A ZA 2018/03956A ZA 201803956 A ZA201803956 A ZA 201803956A ZA 201803956 B ZA201803956 B ZA 201803956B
- Authority
- ZA
- South Africa
- Prior art keywords
- adeno
- treatment
- viral vectors
- spinal muscular
- associated viral
- Prior art date
Links
- 230000003387 muscular Effects 0.000 title 1
- 239000013603 viral vector Substances 0.000 title 1
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
- 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
-
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- 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
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- 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
- C07K14/4702—Regulators; Modulating activity
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
- C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
- C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
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)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Toxicology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Marine Sciences & Fisheries (AREA)
- Immunology (AREA)
- Neurosurgery (AREA)
- Psychology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267012P | 2015-12-14 | 2015-12-14 | |
| PCT/US2016/066669 WO2017106354A1 (en) | 2015-12-14 | 2016-12-14 | Adeno-associated viral vectors useful in treatment of spinal muscular atropy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ZA201803956B true ZA201803956B (en) | 2019-04-24 |
Family
ID=57737990
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ZA2018/03956A ZA201803956B (en) | 2015-12-14 | 2018-06-13 | Adeno-associated viral vectors useful in treatment of spinal muscular atropy |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US20180353624A1 (enExample) |
| EP (1) | EP3394270A1 (enExample) |
| JP (1) | JP7082050B2 (enExample) |
| KR (1) | KR20180086266A (enExample) |
| CN (1) | CN109072254A (enExample) |
| AU (1) | AU2016370630B2 (enExample) |
| BR (1) | BR112018011975A2 (enExample) |
| CA (1) | CA3008280A1 (enExample) |
| IL (1) | IL259877A (enExample) |
| MA (1) | MA44119A (enExample) |
| MX (1) | MX2018007234A (enExample) |
| WO (1) | WO2017106354A1 (enExample) |
| ZA (1) | ZA201803956B (enExample) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG10202100632WA (en) * | 2016-07-26 | 2021-03-30 | Univ Cornell | Gene therapy for the treatment of aldehyde dehydrogenase deficiency |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| US11827906B2 (en) | 2017-02-28 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clade f vector and uses therefor |
| AU2018320849A1 (en) * | 2017-08-25 | 2020-03-05 | Ovid Therapeutics Inc. | Recombinant adeno-associated vectors |
| JP2021534154A (ja) * | 2018-08-15 | 2021-12-09 | バイオジェン・エムエイ・インコーポレイテッドBiogen MA Inc. | 脊髄性筋萎縮症のための併用療法 |
| WO2020127813A1 (en) * | 2018-12-21 | 2020-06-25 | Genethon | Expression cassettes for gene therapy vectors |
| EP4013387A4 (en) * | 2019-08-15 | 2023-09-27 | Biogen MA Inc. | COMBINATION THERAPY FOR SPINAL MUSCLE ATROPHY |
| CN112011571A (zh) * | 2020-04-26 | 2020-12-01 | 辉大(上海)生物科技有限公司 | 一种用于治疗脊髓性肌萎缩的基因治疗药物 |
| EP4142800A1 (en) * | 2020-04-28 | 2023-03-08 | Genethon | Use of a synthetic aav capsid for gene therapy of muscle and central nervous system disorders |
| AU2021270447A1 (en) | 2020-05-12 | 2023-01-05 | The Trustees Of The University Of Pennsylvania | Compositions for DRG-specific reduction of transgene expression |
| RU2742837C1 (ru) * | 2020-06-02 | 2021-02-11 | Общество С Ограниченной Ответственностью "Анабион" | Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение |
| CN113755524B (zh) * | 2020-06-02 | 2023-11-03 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| CN112852882A (zh) * | 2021-02-04 | 2021-05-28 | 中吉智药(南京)生物技术有限公司 | 一种杆状病毒感染昆虫细胞生产aav基因药物的系统及方法 |
| EP4430170A2 (en) * | 2021-11-09 | 2024-09-18 | Asimov, Inc. | Stable production systems for aav vector production |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| KR102717399B1 (ko) * | 2022-05-10 | 2024-10-16 | 서울대학교산학협력단 | 인간 smn1 단백질 변이체 및 이의 용도 |
| WO2024165839A1 (en) * | 2023-02-06 | 2024-08-15 | Royal Holloway And Bedford New College | Transgenes |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2298926A1 (en) * | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| CN104704123A (zh) * | 2012-06-21 | 2015-06-10 | 肌肉学研究协会 | 基因治疗载体的广泛的基因递送 |
| AU2013388083B2 (en) * | 2013-05-01 | 2019-08-22 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
| US20150110857A1 (en) * | 2013-10-22 | 2015-04-23 | Shire Human Genetic Therapies, Inc. | Cns delivery of mrna and uses thereof |
| GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| LT3872085T (lt) * | 2015-07-30 | 2023-05-10 | Massachusetts Eye & Ear Infirmary | Viruso prototipo sekos ir jų panaudojimas |
-
2016
- 2016-12-14 JP JP2018531163A patent/JP7082050B2/ja active Active
- 2016-12-14 WO PCT/US2016/066669 patent/WO2017106354A1/en not_active Ceased
- 2016-12-14 MX MX2018007234A patent/MX2018007234A/es unknown
- 2016-12-14 BR BR112018011975A patent/BR112018011975A2/pt not_active Application Discontinuation
- 2016-12-14 CN CN201680081819.2A patent/CN109072254A/zh active Pending
- 2016-12-14 EP EP16822581.1A patent/EP3394270A1/en not_active Withdrawn
- 2016-12-14 AU AU2016370630A patent/AU2016370630B2/en not_active Expired - Fee Related
- 2016-12-14 MA MA044119A patent/MA44119A/fr unknown
- 2016-12-14 US US16/061,109 patent/US20180353624A1/en not_active Abandoned
- 2016-12-14 KR KR1020187019828A patent/KR20180086266A/ko not_active Withdrawn
- 2016-12-14 CA CA3008280A patent/CA3008280A1/en active Pending
-
2018
- 2018-06-07 IL IL259877A patent/IL259877A/en unknown
- 2018-06-13 ZA ZA2018/03956A patent/ZA201803956B/en unknown
-
2021
- 2021-09-30 US US17/490,611 patent/US20220265861A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| CN109072254A (zh) | 2018-12-21 |
| JP7082050B2 (ja) | 2022-06-07 |
| CA3008280A1 (en) | 2017-06-22 |
| AU2016370630B2 (en) | 2023-04-13 |
| BR112018011975A2 (pt) | 2018-12-11 |
| IL259877A (en) | 2018-07-31 |
| KR20180086266A (ko) | 2018-07-30 |
| US20220265861A1 (en) | 2022-08-25 |
| WO2017106354A1 (en) | 2017-06-22 |
| US20180353624A1 (en) | 2018-12-13 |
| MA44119A (fr) | 2018-10-31 |
| JP2018537984A (ja) | 2018-12-27 |
| EP3394270A1 (en) | 2018-10-31 |
| AU2016370630A1 (en) | 2018-06-28 |
| MX2018007234A (es) | 2018-11-09 |
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