CA3008280A1 - Adeno-associated viral vectors useful in treatment of spinal muscular atropy - Google Patents
Adeno-associated viral vectors useful in treatment of spinal muscular atropy Download PDFInfo
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- CA3008280A1 CA3008280A1 CA3008280A CA3008280A CA3008280A1 CA 3008280 A1 CA3008280 A1 CA 3008280A1 CA 3008280 A CA3008280 A CA 3008280A CA 3008280 A CA3008280 A CA 3008280A CA 3008280 A1 CA3008280 A1 CA 3008280A1
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Classifications
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- 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
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- 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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- 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
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- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
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- 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
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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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2800/00—Nucleic acids vectors
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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| US201562267012P | 2015-12-14 | 2015-12-14 | |
| US62/267,012 | 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 |
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| US (2) | US20180353624A1 (enExample) |
| EP (1) | EP3394270A1 (enExample) |
| JP (1) | JP7082050B2 (enExample) |
| KR (1) | KR20180086266A (enExample) |
| CN (1) | CN109072254A (enExample) |
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| MX (1) | MX2018007234A (enExample) |
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| 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 |
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| 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 |
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- 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 |
| ZA201803956B (en) | 2019-04-24 |
| JP7082050B2 (ja) | 2022-06-07 |
| 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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