JP2018537984A5 - - Google Patents
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- Publication number
- JP2018537984A5 JP2018537984A5 JP2018531163A JP2018531163A JP2018537984A5 JP 2018537984 A5 JP2018537984 A5 JP 2018537984A5 JP 2018531163 A JP2018531163 A JP 2018531163A JP 2018531163 A JP2018531163 A JP 2018531163A JP 2018537984 A5 JP2018537984 A5 JP 2018537984A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- aav
- composition
- sequence
- vector according
- Prior art date
- Legal status (The legal status 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 status listed.)
- Granted
Links
- 239000013598 vector Substances 0.000 claims 14
- 239000000203 mixture Substances 0.000 claims 12
- 239000013607 AAV vector Substances 0.000 claims 11
- 210000000234 capsid Anatomy 0.000 claims 4
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 150000007523 nucleic acids Chemical group 0.000 claims 3
- 239000013603 viral vector Substances 0.000 claims 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 2
- 102100021947 Survival motor neuron protein Human genes 0.000 claims 2
- 210000004027 cell Anatomy 0.000 claims 2
- 208000002320 spinal muscular atrophy Diseases 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 108020004705 Codon Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 claims 1
- 241000124008 Mammalia Species 0.000 claims 1
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- 125000002924 primary amino group Chemical group [H]N([H])* 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018537984A JP2018537984A (ja) | 2018-12-27 |
| JP2018537984A5 true JP2018537984A5 (OSRAM) | 2020-01-23 |
| JP7082050B2 JP7082050B2 (ja) | 2022-06-07 |
Family
ID=57737990
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018531163A Active JP7082050B2 (ja) | 2015-12-14 | 2016-12-14 | 脊髄性筋萎縮症の処置において有用なアデノ－関連ウイルスベクター |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US20180353624A1 (OSRAM) |
| EP (1) | EP3394270A1 (OSRAM) |
| JP (1) | JP7082050B2 (OSRAM) |
| KR (1) | KR20180086266A (OSRAM) |
| CN (1) | CN109072254A (OSRAM) |
| AU (1) | AU2016370630B2 (OSRAM) |
| BR (1) | BR112018011975A2 (OSRAM) |
| CA (1) | CA3008280A1 (OSRAM) |
| IL (1) | IL259877A (OSRAM) |
| MA (1) | MA44119A (OSRAM) |
| MX (1) | MX2018007234A (OSRAM) |
| WO (1) | WO2017106354A1 (OSRAM) |
| ZA (1) | ZA201803956B (OSRAM) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7165357B2 (ja) * | 2016-07-26 | 2022-11-04 | コーネル ユニヴァーシティー | アルデヒドデヒドロゲナーゼ欠損症の治療のための遺伝子治療 |
| WO2018160582A1 (en) | 2017-02-28 | 2018-09-07 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) clade f vector and uses therefor |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| 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 |
| EP3898995A1 (en) * | 2018-12-21 | 2021-10-27 | Genethon | Expression cassettes for gene therapy vectors |
| JP7689110B2 (ja) * | 2019-08-15 | 2025-06-05 | バイオジェン・エムエイ・インコーポレイテッド | 脊髄性筋萎縮症のための併用療法 |
| 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 |
| WO2021219762A1 (en) * | 2020-04-28 | 2021-11-04 | Genethon | Use of a synthetic aav capsid for gene therapy of muscle and central nervous system disorders |
| KR20230010670A (ko) | 2020-05-12 | 2023-01-19 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 이식유전자 발현의 drg-특이적 감소를 위한 조성물 |
| 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 |
| RU2742837C1 (ru) * | 2020-06-02 | 2021-02-11 | Общество С Ограниченной Ответственностью "Анабион" | Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение |
| CA3209779A1 (en) | 2021-02-01 | 2022-08-04 | 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 |
| WO2023086822A2 (en) * | 2021-11-09 | 2023-05-19 | 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 |
|---|---|---|---|---|
| EP2292780B1 (en) * | 2003-09-30 | 2017-08-23 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| EP3536795A3 (en) * | 2012-06-21 | 2019-12-11 | Association Institut de Myologie | Widespread gene expression |
| KR102116378B1 (ko) * | 2013-05-01 | 2020-06-01 | 젠자임 코포레이션 | 척수성 근위축증을 치료하기 위한 조성물 및 방법 |
| CA2928040A1 (en) | 2013-10-22 | 2015-04-30 | 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 |
| JP7166168B2 (ja) * | 2015-07-30 | 2022-11-07 | マサチューセッツ アイ アンド イヤー インファーマリー | 祖先ウイルス配列およびその使用 |
-
2016
- 2016-12-14 EP EP16822581.1A patent/EP3394270A1/en not_active Withdrawn
- 2016-12-14 CN CN201680081819.2A patent/CN109072254A/zh active Pending
- 2016-12-14 MA MA044119A patent/MA44119A/fr unknown
- 2016-12-14 KR KR1020187019828A patent/KR20180086266A/ko not_active Withdrawn
- 2016-12-14 BR BR112018011975A patent/BR112018011975A2/pt not_active Application Discontinuation
- 2016-12-14 US US16/061,109 patent/US20180353624A1/en not_active Abandoned
- 2016-12-14 MX MX2018007234A patent/MX2018007234A/es unknown
- 2016-12-14 CA CA3008280A patent/CA3008280A1/en active Pending
- 2016-12-14 WO PCT/US2016/066669 patent/WO2017106354A1/en not_active Ceased
- 2016-12-14 AU AU2016370630A patent/AU2016370630B2/en not_active Expired - Fee Related
- 2016-12-14 JP JP2018531163A patent/JP7082050B2/ja active Active
-
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
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