SA517382162B1 - طرق وتركيبات لمعالجة مرض بصري وراثي - Google Patents
طرق وتركيبات لمعالجة مرض بصري وراثي Download PDFInfo
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Families Citing this family (57)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| JP7046828B2 (ja) | 2016-04-15 | 2022-04-04 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 滲出型加齢性黄斑変性の治療のための組成物 |
| EP3827812B1 (en) | 2016-07-29 | 2025-10-29 | The Regents of the University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| WO2018045347A1 (en) * | 2016-09-02 | 2018-03-08 | Spark Therapeutics, Inc. | Methods and vectors for treating cns disorders |
| WO2019006182A1 (en) * | 2017-06-30 | 2019-01-03 | The Regents Of The University Of California | VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF |
| JP7221275B2 (ja) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Aavを送達するための組成物および方法 |
| JP2020534788A (ja) | 2017-08-28 | 2020-12-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | アデノ随伴ウイルスカプシド変異体及びその使用方法 |
| NZ763617A (en) * | 2017-10-16 | 2026-01-30 | Vigeneron Gmbh | Aav vectors |
| SG11202003778QA (en) * | 2017-11-13 | 2020-05-28 | Univ California | Compositions and methods for enhancing visual function |
| MA50877A (fr) | 2017-11-21 | 2020-09-30 | Bayer Healthcare Llc | Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante |
| CN112105630A (zh) * | 2018-02-15 | 2020-12-18 | T·比约尔克隆德 | 经修饰的病毒衣壳 |
| US12558434B2 (en) | 2018-02-20 | 2026-02-24 | The Trustees Of The University Of Pennsylvania | Compositions for treatment of wet age-related macular degeneration |
| US12421525B2 (en) | 2018-02-28 | 2025-09-23 | University of Pittsburgh—of the Commonwealth System of Higher Education | Conjugates comprising AAVs and CAS9 polypeptides |
| SG11202011296VA (en) | 2018-05-15 | 2020-12-30 | Voyager Therapeutics Inc | Compositions and methods for the treatment of parkinson's disease |
| TW202015742A (zh) | 2018-05-15 | 2020-05-01 | 美商航海家醫療公司 | 投遞腺相關病毒(aav)之組成物和方法 |
| US12060567B2 (en) | 2018-06-13 | 2024-08-13 | Voyager Therapeutics, Inc. | Engineered untranslated regions (UTR) for AAV production |
| AU2019310459A1 (en) | 2018-07-24 | 2021-02-18 | Voyager Therapeutics, Inc. | Systems and methods for producing gene therapy formulations |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| WO2020072849A1 (en) | 2018-10-04 | 2020-04-09 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| WO2020072844A1 (en) | 2018-10-05 | 2020-04-09 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding aav production proteins |
| WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| CA3116701A1 (en) | 2018-10-15 | 2020-04-23 | Voyager Therapeutics, Inc. | Expression vectors for large-scale production of raav in the baculovirus/sf9 system |
| JP2022522995A (ja) | 2019-01-18 | 2022-04-21 | ボイジャー セラピューティクス インコーポレイテッド | Aav粒子を生成するための方法及びシステム |
| EP4667578A3 (en) * | 2019-04-03 | 2026-04-01 | REGENXBIO Inc. | Gene therapy for eye pathologies |
| KR20210148333A (ko) * | 2019-04-08 | 2021-12-07 | 더 칠드런스 호스피탈 오브 필라델피아 | Tpp1을 발현하는 aav의 투여를 통한 눈의 리소좀 축적 질환 치료 |
| EP3962536A1 (en) | 2019-04-29 | 2022-03-09 | Voyager Therapeutics, Inc. | Systems and methods for producing baculoviral infected insect cells (biics) in bioreactors |
| WO2020227515A1 (en) | 2019-05-07 | 2020-11-12 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
| WO2021030125A1 (en) | 2019-08-09 | 2021-02-18 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| TW202122582A (zh) | 2019-08-26 | 2021-06-16 | 美商航海家醫療公司 | 病毒蛋白之控制表現 |
| US20220333133A1 (en) | 2019-09-03 | 2022-10-20 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
| CN114729009A (zh) * | 2019-10-31 | 2022-07-08 | 伯尔尼大学 | 用于眼部基因递送的aav载体变体 |
| 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 |
| WO2021207500A2 (en) * | 2020-04-08 | 2021-10-14 | President And Fellows Of Harvard College | TGFβ THERAPY FOR OCULAR AND NEURODEGENERATIVE DISEASES |
| CN116096734A (zh) | 2020-05-13 | 2023-05-09 | 沃雅戈治疗公司 | Aav衣壳的向性的重定向 |
| WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
| JP7842082B2 (ja) * | 2020-07-21 | 2026-04-07 | エフティージェン コーポレーション | 眼疾患を処置するための組成物および方法 |
| US20230285596A1 (en) | 2020-07-27 | 2023-09-14 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
| BR112023001456A2 (pt) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase |
| WO2022032153A1 (en) | 2020-08-06 | 2022-02-10 | Voyager Therapeutics, Inc. | Cell culture medium for use in producing gene therapy products in bioreactors |
| US20240141377A1 (en) | 2021-03-03 | 2024-05-02 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| US20240141378A1 (en) | 2021-03-03 | 2024-05-02 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| CN113121655B (zh) * | 2021-04-19 | 2021-11-19 | 上海信致医药科技有限公司 | 眼部和肌肉特异靶向型腺相关病毒载体及其应用 |
| MX2023012722A (es) * | 2021-04-27 | 2023-11-08 | Massachusetts Gen Hospital | Capsides de aav y usos de las mismas. |
| US20240382617A1 (en) * | 2021-09-21 | 2024-11-21 | Capsida Biotherapeutics, Inc. | Virus compositions to efficiently target the nervous system and the lungs |
| CA3238867A1 (en) | 2021-11-29 | 2023-06-01 | Shanghai Regenelead Therapies Co., Ltd. | Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease |
| CN114195859B (zh) * | 2021-12-10 | 2022-11-18 | 和元生物技术(上海)股份有限公司 | 一种适用于特异感染u251细胞的腺相关病毒突变体 |
| EP4479414A1 (en) | 2022-02-17 | 2024-12-25 | Skyline Therapeutics Limited | Recombinant adeno-associated virus with modified aav capsid polypeptides |
| CN118660913A (zh) | 2022-02-21 | 2024-09-17 | 上海瑞宏迪医药有限公司 | Vegf结合分子及其医药用途 |
| CN119894920A (zh) | 2022-07-22 | 2025-04-25 | 上海天泽云泰生物医药有限公司 | 用于靶向神经系统的新型aav衣壳及其用途 |
| US20260085328A1 (en) | 2022-09-08 | 2026-03-26 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
| EP4688808A1 (en) * | 2023-04-04 | 2026-02-11 | Children's Medical Research Institute | Aav capsids and vectors for transduction of cells of the peripheral nervous system |
| WO2025040166A1 (zh) | 2023-08-23 | 2025-02-27 | 上海瑞宏迪医药有限公司 | 药物组合物及其用途 |
| WO2025214404A1 (en) | 2024-04-10 | 2025-10-16 | Skyline Therapeutics (Shanghai) Co., Ltd. | Optogenetic modulation for vision restoration |
| WO2025214408A1 (en) | 2024-04-10 | 2025-10-16 | Skyline Therapeutics (Shanghai) Co., Ltd. | Optogenetic modulation for vision restoration |
| WO2026005189A1 (ko) * | 2024-06-27 | 2026-01-02 | 이엔셀 주식회사 | 안구 세포 또는 조직을 표적하는 펩타이드 및 이를 포함하는 바이러스 캡시드 단백질 및 바이러스 벡터 |
| KR102901502B1 (ko) * | 2024-06-27 | 2025-12-18 | 이엔셀 주식회사 | 안구 세포 또는 조직을 표적하는 펩타이드 및 이를 포함하는 바이러스 캡시드 단백질 및 바이러스 벡터 |
| CN119039401B (zh) * | 2024-08-19 | 2025-04-11 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6329501B1 (en) * | 1997-05-29 | 2001-12-11 | Auburn University | Methods and compositions for targeting compounds to muscle |
| US6399575B1 (en) * | 1998-11-10 | 2002-06-04 | Auburn University | Methods and compositions for targeting compounds to the central nervous system |
| US6943153B1 (en) * | 1999-03-15 | 2005-09-13 | The Regents Of The University Of California | Use of recombinant gene delivery vectors for treating or preventing diseases of the eye |
| AU2002248297A1 (en) * | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US20100081707A1 (en) * | 2008-02-21 | 2010-04-01 | Ali Robin R | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| US20090215879A1 (en) * | 2008-02-26 | 2009-08-27 | University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
| KR101154374B1 (ko) * | 2009-02-18 | 2012-07-09 | 재단법인 아산사회복지재단 | 표적화된 유전자 전달을 위한 아데노-부속 바이러스 혈청형5 벡터를 포함하는 조성물 |
| US20110052666A1 (en) * | 2009-09-03 | 2011-03-03 | Medtronic, Inc. | Compositions, Methods, and Systems for SIRNA Delivery |
| HRP20171334T1 (hr) * | 2011-04-22 | 2017-11-17 | The Regents Of The University Of California | Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe |
| WO2013159036A1 (en) * | 2012-04-19 | 2013-10-24 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| US9567376B2 (en) * | 2013-02-08 | 2017-02-14 | The Trustees Of The University Of Pennsylvania | Enhanced AAV-mediated gene transfer for retinal therapies |
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| NZ735344A (en) | 2024-09-27 |
| AU2016219789B2 (en) | 2021-03-25 |
| ZA201705628B (en) | 2024-08-28 |
| WO2016134375A1 (en) | 2016-08-25 |
| CN108347932A (zh) | 2018-07-31 |
| SG11201706755SA (en) | 2017-09-28 |
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