JP2022515338A5 - - Google Patents
Info
- Publication number
- JP2022515338A5 JP2022515338A5 JP2021532368A JP2021532368A JP2022515338A5 JP 2022515338 A5 JP2022515338 A5 JP 2022515338A5 JP 2021532368 A JP2021532368 A JP 2021532368A JP 2021532368 A JP2021532368 A JP 2021532368A JP 2022515338 A5 JP2022515338 A5 JP 2022515338A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- promoter
- nucleic acid
- aav
- amino acid
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024177032A JP2025011200A (ja) | 2018-12-05 | 2024-10-09 | 遺伝子送達のための組み換えアデノ随伴ウイルスベクター |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862775871P | 2018-12-05 | 2018-12-05 | |
| US62/775,871 | 2018-12-05 | ||
| US201962801195P | 2019-02-05 | 2019-02-05 | |
| US62/801,195 | 2019-02-05 | ||
| US201962863126P | 2019-06-18 | 2019-06-18 | |
| US62/863,126 | 2019-06-18 | ||
| US201962914856P | 2019-10-14 | 2019-10-14 | |
| US62/914,856 | 2019-10-14 | ||
| PCT/US2019/064396 WO2020117898A1 (en) | 2018-12-05 | 2019-12-04 | Recombinant adeno-associated viral vector for gene delivery |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024177032A Division JP2025011200A (ja) | 2018-12-05 | 2024-10-09 | 遺伝子送達のための組み換えアデノ随伴ウイルスベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022515338A JP2022515338A (ja) | 2022-02-18 |
| JP2022515338A5 true JP2022515338A5 (https=) | 2022-12-12 |
| JP7590968B2 JP7590968B2 (ja) | 2024-11-27 |
Family
ID=70974857
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021532368A Active JP7590968B2 (ja) | 2018-12-05 | 2019-12-04 | 遺伝子送達のための組み換えアデノ随伴ウイルスベクター |
| JP2024177032A Pending JP2025011200A (ja) | 2018-12-05 | 2024-10-09 | 遺伝子送達のための組み換えアデノ随伴ウイルスベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024177032A Pending JP2025011200A (ja) | 2018-12-05 | 2024-10-09 | 遺伝子送達のための組み換えアデノ随伴ウイルスベクター |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US12454701B2 (https=) |
| EP (1) | EP3890786A4 (https=) |
| JP (2) | JP7590968B2 (https=) |
| KR (1) | KR20220022107A (https=) |
| CN (1) | CN113423434A (https=) |
| AU (1) | AU2019391042A1 (https=) |
| BR (1) | BR112021009913A2 (https=) |
| CA (1) | CA3121177A1 (https=) |
| IL (1) | IL283546A (https=) |
| MX (1) | MX2021006646A (https=) |
| WO (1) | WO2020117898A1 (https=) |
Families Citing this family (51)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK3635009T3 (da) | 2017-06-07 | 2026-03-30 | Regeneron Pharma | Sammensætninger og fremgangsmåder til internalisering af enzymer |
| US12258597B2 (en) | 2018-02-07 | 2025-03-25 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for therapeutic protein delivery |
| BR112020023145A2 (pt) | 2018-05-17 | 2021-02-02 | Regeneron Pharmaceuticals, Inc. | anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto |
| CA3114175A1 (en) | 2018-10-01 | 2020-04-09 | The Trustees Of The University Of Pennsylvania | Beta-galactosidase based aav for treating gm1 gangliosidosis |
| KR20220022107A (ko) * | 2018-12-05 | 2022-02-24 | 아베오나 테라퓨틱스 인코퍼레이티드 | 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터 |
| CA3132379A1 (en) * | 2019-03-22 | 2020-10-01 | Rush University Medical Center | Combination of nasal gene delivery and oral cinnamic acid, oleamide or gemfibrozil for lysosomal storage disorders |
| US20210082541A1 (en) * | 2019-08-31 | 2021-03-18 | Wyatt Technology Corporation | Measuring attributes of a viral gene delivery vehicle sample via separation |
| CN113025618B (zh) * | 2019-12-24 | 2024-02-06 | 朗信启昇(苏州)生物制药有限公司 | 一种x连锁遗传性视网膜劈裂症的基因治疗方案与应用 |
| EP4103214B1 (en) | 2020-02-13 | 2026-04-29 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| WO2021163357A2 (en) | 2020-02-13 | 2021-08-19 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| WO2021183895A1 (en) * | 2020-03-13 | 2021-09-16 | Biomarin Pharmaceutical Inc. | Treatment of fabry disease with aav gene therapy vectors |
| AU2021265768B2 (en) * | 2020-04-27 | 2023-03-30 | 4D Molecular Therapeutics Inc. | Adeno-associated variants, formulations and methods for pulmonary delivery |
| EP4103211A4 (en) * | 2020-04-27 | 2023-08-23 | 4D Molecular Therapeutics Inc. | CODON-OPTIMISED GLA GENES AND THEIR USES |
| GB202010981D0 (en) * | 2020-07-16 | 2020-09-02 | Ucl Business Ltd | Gene therapy for neuromuscular and neuromotor disorders |
| KR20230117327A (ko) * | 2020-09-03 | 2023-08-08 | 어빈 에스.와이. 첸 | 가용성 알칼리성 포스파타제 작제물 및 가용성 알칼리성 포스파타제 작제물을 인코딩하는 폴리뉴클레오티드를 포함하는 발현 벡터 |
| CN114507692B (zh) * | 2020-11-16 | 2024-07-12 | 舒泰神（北京）生物制药股份有限公司 | 用于治疗法布里病的腺相关病毒载体及其用途 |
| EP4244360A4 (en) * | 2020-11-16 | 2025-05-07 | Beijing SoloBio Genetechnology Company Ltd. | Recombinant adeno-associated viruses with enhanced liver tropism and uses thereof |
| MX2023008362A (es) * | 2021-01-14 | 2023-10-04 | Spark Therapeutics Inc | Composiciones y metodos para el tratamiento de la enfermedad de fabry. |
| AR124981A1 (es) * | 2021-02-26 | 2023-05-24 | Takeda Pharmaceuticals Co | Composición y métodos para el tratamiento de la enfermedad de fabry |
| JP2024519819A (ja) * | 2021-05-18 | 2024-05-21 | アビオナ・セラピュ―ティクス・インコーポレイテッド | 眼疾患および障害を処置するための方法および組成物 |
| KR20240032971A (ko) | 2021-07-08 | 2024-03-12 | 테나야 테라퓨틱스, 인코포레이티드 | 유전자 요법을 위한 최적화된 발현 카세트 |
| WO2023284873A1 (en) * | 2021-07-15 | 2023-01-19 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant adeno-associated viral vectors for treating bietti crystalline dystrophy |
| AR126877A1 (es) * | 2021-08-25 | 2023-11-22 | Canbridge Pharmaceuticals Inc | PARTÍCULAS DE AAV QUE COMPRENDEN UNA PROTEÍNA DE LA CÁPSIDE HEPATOTRÓPICA Y a-GLUCOSIDASA ÁCIDA (GAA) Y SU USO PARA TRATAR LA ENFERMEDAD DE POMPE |
| CA3229998A1 (en) * | 2021-08-25 | 2023-03-02 | Canbridge Pharmaceuticals, Inc. | Aav particles comprising a liver-tropic capsid protein and alpha-galactosidase and their use to treat fabry disease |
| CN114181318B (zh) * | 2021-11-08 | 2023-08-01 | 四川大学 | 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用 |
| KR20240133693A (ko) * | 2021-11-12 | 2024-09-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 점액다당류증 iiia의 치료를 위한 유전자 요법 |
| CN114381465B (zh) * | 2021-12-22 | 2024-01-16 | 苏州诺洁贝生物技术有限公司 | 优化的cyp4v2基因及其用途 |
| EP4511482A4 (en) * | 2022-04-19 | 2026-03-18 | Shanghai Vitalgen Biopharma Co Ltd | Recombinant AAV vectors for the treatment of neurodegenerative disorders |
| 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 |
| WO2023240236A1 (en) * | 2022-06-10 | 2023-12-14 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of spinal muscular atrophy related disorders |
| US20250369012A1 (en) * | 2022-06-21 | 2025-12-04 | Skyline Therapeutics Limited | Recombinant aav for the gene therapy of sma disease |
| KR20240000814A (ko) * | 2022-06-24 | 2024-01-03 | 연세대학교 산학협력단 | 망막색소상피에 특이적으로 작동하는 CRISPR/Cas 복합체를 유효성분으로 포함하는 망막질환 치료용 약학조성물 |
| CA3262218A1 (en) | 2022-07-19 | 2024-01-25 | Rampart Bioscience, Inc. | CIRCULAR NON-IMMUNOGENEOUS NON-VIRAL DNA VECTORS |
| WO2024054864A1 (en) | 2022-09-06 | 2024-03-14 | Tenaya Therapeutics, Inc. | Cardioprotective heart disease therapies |
| CN118715316B (zh) * | 2022-10-08 | 2025-09-19 | 凌意(杭州)生物科技有限公司 | 用于治疗GCase缺乏相关疾病的多核苷酸 |
| CN115960177B (zh) * | 2022-10-09 | 2023-07-07 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| KR20240086741A (ko) * | 2022-11-28 | 2024-06-19 | (주)지뉴인텍 | Pde6b 유전자가 포함된 아데노연관바이러스 재조합 벡터를 유효성분으로 포함하는 유전성 망막질환 예방 또는 치료용 조성물 |
| IT202300004443A1 (it) * | 2023-03-09 | 2024-09-09 | Int Centre For Genetic Engineering And Biotechnology Icgeb | Sequenza codificante per alfa galattosidasi a umana per il trattamento della malattia di fabry |
| WO2024215655A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Cardioprotective bag3 therapies |
| WO2024215653A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Guide rnas, vectors, and virions for targeting mutations in the pln gene |
| AU2024257162A1 (en) * | 2023-04-17 | 2025-11-06 | Exegenesis Bio Singapore Pte. Ltd. | Compositions and methods for gene therapies |
| EP4704920A1 (en) * | 2023-05-04 | 2026-03-11 | Abeona Therapeutics Inc. | Recombinant adeno-associated viral vectors for multipartite gene delivery and stargardt disease treatment |
| WO2024233669A2 (en) * | 2023-05-11 | 2024-11-14 | Saliogen Therapeutics, Inc. | Compositions and methods for treatment of cystic fibrosis |
| EP4711461A1 (en) * | 2023-05-12 | 2026-03-18 | Genecombio Ltd. | Viral vector carrying smn gene expression cassette and use thereof |
| CN116622750B (zh) * | 2023-06-01 | 2024-05-31 | 上海勉亦生物科技有限公司 | 优化的人法布里转基因表达盒及其用途 |
| CN121548647A (zh) * | 2023-06-09 | 2026-02-17 | 4D分子治疗有限公司 | 腺伴随病毒制剂 |
| WO2025030426A1 (en) * | 2023-08-09 | 2025-02-13 | Chigenovo Co., Ltd. | Pharmaceutical preparations for treating bietti's crystalline dystrophy |
| WO2025039622A1 (zh) * | 2023-08-23 | 2025-02-27 | 上海金珂博生物技术有限公司 | 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途 |
| WO2025090786A1 (en) * | 2023-10-24 | 2025-05-01 | Rampart Bioscience, Inc. | Reduced immunogenic gene therapy protocols using circular, extended cruciform containing non-viral vectors |
| WO2025092951A1 (en) * | 2023-11-03 | 2025-05-08 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant adeno-associated viral vectors for treating fabry disease |
| WO2026072721A1 (en) * | 2024-09-25 | 2026-04-02 | Carbon Biosciences, Inc. | Parvovirus compositions and methods for gene therapy |
Family Cites Families (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SE9101433D0 (sv) | 1991-05-13 | 1991-05-13 | Marianne Hansson | Recombinant dna sequence and its use |
| US6582692B1 (en) | 1999-11-17 | 2003-06-24 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
| US7638120B2 (en) | 2000-03-14 | 2009-12-29 | Thomas Jefferson University | High transgene expression of a pseudotyped adeno-associated virus type |
| 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 |
| US20030083299A1 (en) | 2000-11-04 | 2003-05-01 | Ferguson Ian A. | Non-invasive delivery of polypeptides through the blood-brain barrier |
| DE10120265A1 (de) | 2001-04-25 | 2002-11-14 | Deutsches Krebsforsch | AAV-Helferplasmide zur Helfervirus-freien Verpackung und Pseudotypisierung von AAV-Vektoren |
| DK2573170T3 (en) | 2001-12-17 | 2018-04-09 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use |
| EP1620133B1 (en) | 2003-05-01 | 2015-12-09 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| US7527947B2 (en) | 2004-06-14 | 2009-05-05 | Novozymes A/S | Signal peptide for producing a polypeptide |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| US7867484B2 (en) | 2006-01-27 | 2011-01-11 | University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| CN103849629B (zh) | 2006-06-21 | 2017-06-09 | 尤尼克尔Ip股份有限公司 | 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体 |
| CA2665620A1 (en) * | 2006-10-12 | 2008-04-17 | Copernicus Therapeutics Inc. | Codon optimized cftr |
| EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
| EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| WO2009137006A2 (en) | 2008-04-30 | 2009-11-12 | The University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| US9415121B2 (en) | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| EP2561073B1 (en) | 2010-04-23 | 2016-08-24 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| WO2012057363A1 (ja) | 2010-10-27 | 2012-05-03 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| CN105755044A (zh) * | 2011-04-22 | 2016-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| IL287381B2 (en) * | 2011-11-14 | 2024-04-01 | Astellas Inst For Regenerative Medicine | Pharmaceutical preparations of human rpe cells and uses thereof |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| WO2013174760A1 (en) | 2012-05-25 | 2013-11-28 | Deutsches Krebsforschungszentrum | Optimized aav-vectors for high transduction rates in dendritic cells |
| EP2900686B1 (en) | 2012-09-28 | 2020-06-10 | The University of North Carolina At Chapel Hill | Aav vectors targeted to oligodendrocytes |
| EP2840140B2 (en) | 2012-12-12 | 2023-02-22 | The Broad Institute, Inc. | Crispr-Cas based method for mutation of prokaryotic cells |
| AU2014255665B2 (en) * | 2013-04-18 | 2018-08-02 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| EP4344741A3 (en) | 2014-11-21 | 2024-08-28 | The University of North Carolina at Chapel Hill | Aav vectors targeted to the central nervous system |
| CA3012344A1 (en) | 2016-02-12 | 2017-08-17 | University Of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
| IL305149A (en) * | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| WO2018178067A1 (en) * | 2017-03-27 | 2018-10-04 | Vrije Universiteit Brussel | Diaphragm-specific nucleic acid regulatory elements and methods and use thereof |
| JP7371083B2 (ja) | 2018-07-20 | 2023-10-30 | ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ | 遺伝子治療発現ベクターの眼内送達 |
| KR20220022107A (ko) | 2018-12-05 | 2022-02-24 | 아베오나 테라퓨틱스 인코퍼레이티드 | 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터 |
| JP2024519819A (ja) | 2021-05-18 | 2024-05-21 | アビオナ・セラピュ―ティクス・インコーポレイテッド | 眼疾患および障害を処置するための方法および組成物 |
-
2019
- 2019-12-04 KR KR1020217018249A patent/KR20220022107A/ko not_active Ceased
- 2019-12-04 EP EP19892936.6A patent/EP3890786A4/en active Pending
- 2019-12-04 WO PCT/US2019/064396 patent/WO2020117898A1/en not_active Ceased
- 2019-12-04 JP JP2021532368A patent/JP7590968B2/ja active Active
- 2019-12-04 MX MX2021006646A patent/MX2021006646A/es unknown
- 2019-12-04 AU AU2019391042A patent/AU2019391042A1/en not_active Abandoned
- 2019-12-04 CA CA3121177A patent/CA3121177A1/en active Pending
- 2019-12-04 US US17/299,434 patent/US12454701B2/en active Active
- 2019-12-04 CN CN201980088032.2A patent/CN113423434A/zh active Pending
- 2019-12-04 BR BR112021009913-1A patent/BR112021009913A2/pt not_active IP Right Cessation
-
2021
- 2021-05-30 IL IL283546A patent/IL283546A/en unknown
-
2024
- 2024-10-09 JP JP2024177032A patent/JP2025011200A/ja active Pending
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