JPWO2021102234A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2021102234A5 JPWO2021102234A5 JP2022529681A JP2022529681A JPWO2021102234A5 JP WO2021102234 A5 JPWO2021102234 A5 JP WO2021102234A5 JP 2022529681 A JP2022529681 A JP 2022529681A JP 2022529681 A JP2022529681 A JP 2022529681A JP WO2021102234 A5 JPWO2021102234 A5 JP WO2021102234A5
- Authority
- JP
- Japan
- Prior art keywords
- capsid protein
- modified
- seq
- targeting peptide
- aav capsid
- 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 (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025044678A JP2025102825A (ja) | 2019-11-22 | 2025-03-19 | アデノ随伴ウイルスベクター変種 |
| JP2025116303A JP2025148448A (ja) | 2019-11-22 | 2025-07-10 | アデノ随伴ウイルスベクター変種 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962939315P | 2019-11-22 | 2019-11-22 | |
| US62/939,315 | 2019-11-22 | ||
| US202063084709P | 2020-09-29 | 2020-09-29 | |
| US63/084,709 | 2020-09-29 | ||
| PCT/US2020/061464 WO2021102234A1 (en) | 2019-11-22 | 2020-11-20 | Adeno-associated viral vector variants |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025044678A Division JP2025102825A (ja) | 2019-11-22 | 2025-03-19 | アデノ随伴ウイルスベクター変種 |
| JP2025116303A Division JP2025148448A (ja) | 2019-11-22 | 2025-07-10 | アデノ随伴ウイルスベクター変種 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2023503455A JP2023503455A (ja) | 2023-01-30 |
| JPWO2021102234A5 true JPWO2021102234A5 (https=) | 2024-01-30 |
| JP2023503455A5 JP2023503455A5 (https=) | 2024-01-30 |
| JP7712271B2 JP7712271B2 (ja) | 2025-07-23 |
Family
ID=75980202
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022529681A Active JP7712271B2 (ja) | 2019-11-22 | 2020-11-20 | アデノ随伴ウイルスベクター変種 |
| JP2025044678A Pending JP2025102825A (ja) | 2019-11-22 | 2025-03-19 | アデノ随伴ウイルスベクター変種 |
| JP2025116303A Pending JP2025148448A (ja) | 2019-11-22 | 2025-07-10 | アデノ随伴ウイルスベクター変種 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025044678A Pending JP2025102825A (ja) | 2019-11-22 | 2025-03-19 | アデノ随伴ウイルスベクター変種 |
| JP2025116303A Pending JP2025148448A (ja) | 2019-11-22 | 2025-07-10 | アデノ随伴ウイルスベクター変種 |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US20230346981A1 (https=) |
| EP (1) | EP4061427A4 (https=) |
| JP (3) | JP7712271B2 (https=) |
| KR (1) | KR20220116459A (https=) |
| CN (2) | CN115023242B (https=) |
| AU (1) | AU2020388404A1 (https=) |
| BR (1) | BR112022009864A2 (https=) |
| CA (1) | CA3159113A1 (https=) |
| CO (1) | CO2022008507A2 (https=) |
| IL (1) | IL293140A (https=) |
| MX (1) | MX2022006188A (https=) |
| PH (1) | PH12022551247A1 (https=) |
| WO (1) | WO2021102234A1 (https=) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2022013030A (es) * | 2020-04-17 | 2023-03-09 | Regeneron Pharma | Ensayos de deteccion para anticuerpos neutralizantes de coronavirus. |
| MX2024004217A (es) | 2021-10-08 | 2024-06-26 | Dyno Therapeutics Inc | Variantes de cápside y métodos de uso de estas. |
| WO2023205397A1 (en) * | 2022-04-22 | 2023-10-26 | The Children's Hospital Of Philadelphia | Human ependyma-specific promoter and uses thereof |
| AU2023269646A1 (en) * | 2022-05-11 | 2024-12-05 | The Children's Hospital Of Philadelphia | Adeno-associated viral vectors for targeting deep brain structures |
| CN119562959A (zh) * | 2022-05-13 | 2025-03-04 | 费城儿童医院 | 用于靶向脑微脉管系统的腺相关病毒载体 |
| AU2023273809A1 (en) | 2022-05-16 | 2025-01-02 | Genzyme Corporation | Methods of treating metachromatic leukodystrophy |
| 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 |
| KR102870168B1 (ko) * | 2022-08-26 | 2025-10-13 | 김선아 | 활막 표적화 화합물 및 이의 용도 |
| JP2025532627A (ja) * | 2022-09-19 | 2025-10-01 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア | SPLiT-seqを用いたシングルセル解像でのAAV進化 |
| IL322459A (en) * | 2023-01-31 | 2025-09-01 | Childrens Hospital Philadelphia | Innovative gene therapy construct for cln2 disease |
| TW202502803A (zh) | 2023-03-10 | 2025-01-16 | 美商戴諾治療公司 | 衣殼多肽及其使用方法 |
| CN118005749B (zh) * | 2023-07-21 | 2024-07-16 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| TW202525832A (zh) | 2023-08-31 | 2025-07-01 | 美商戴諾治療公司 | 衣殼多肽及其使用方法(二) |
| WO2025090631A1 (en) * | 2023-10-23 | 2025-05-01 | The Children's Hospital Of Philadelphia | Aav vectors for delivery of apoe2 |
| WO2025090598A1 (en) * | 2023-10-23 | 2025-05-01 | The Children's Hospital Of Philadelphia | Aav vectors for treatment of cln2 disease |
| WO2025106874A1 (en) * | 2023-11-15 | 2025-05-22 | Genzyme Corporation | Methods of treating neurodegenerative disorders |
| US20250152747A1 (en) | 2023-11-15 | 2025-05-15 | Genzyme Corporation | Methods of treating gaucher disease and gba-parkinson’s disease |
| TW202545971A (zh) | 2024-02-08 | 2025-12-01 | 美商戴諾治療公司 | 殼體多肽及其使用方法 |
| WO2025213097A1 (en) * | 2024-04-05 | 2025-10-09 | The Children's Hospital Of Philadelphia | Novel gene therapy constructs for stxbp1 haploinsufficiency |
| WO2025231406A1 (en) * | 2024-05-02 | 2025-11-06 | The Children's Hospital Of Philadelphia | Methods to increase transduction of ependyma cells in brain |
| WO2026064442A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| CN120242044B (zh) * | 2025-06-06 | 2025-09-23 | 昆明理工大学 | 一种靶向性的药物递送载体及其制备方法 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20040031072A1 (en) * | 1999-05-06 | 2004-02-12 | La Rosa Thomas J. | Soy nucleic acid molecules and other molecules associated with transcription plants and uses thereof for plant improvement |
| DK2359869T3 (en) * | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| US9585971B2 (en) * | 2013-09-13 | 2017-03-07 | California Institute Of Technology | Recombinant AAV capsid protein |
| DE102014207498A1 (de) * | 2014-04-17 | 2015-10-22 | Universitätsklinikum Hamburg-Eppendorf | Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark |
| KR102423442B1 (ko) * | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| BR112019016769A2 (pt) * | 2017-02-15 | 2020-05-26 | The University Of North Carolina At Chapel Hill | Métodos e composições para transferência genética através da vasculatura |
| MX2019013528A (es) * | 2017-05-12 | 2020-07-14 | The Children´S Hospital Of Philadelphia | Variantes de sulfamidasa (sgsh) y vectores, composiciones, metodos y usos para tratar la mucopolisacaridosis tipo iiia (mps iiia). |
| SG11202002276VA (en) * | 2017-09-20 | 2020-04-29 | 4D Molecular Therapeutics Inc | Adeno-associated virus variant capsids and methods of use thereof |
| US20200263199A1 (en) * | 2017-09-29 | 2020-08-20 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| WO2019113538A2 (en) * | 2017-12-07 | 2019-06-13 | California Institute Of Technology | Methods and systems for noninvasive control of brain cells and related vectors and compositions |
| 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 |
| CN112703198B (zh) * | 2018-07-11 | 2025-05-30 | 布里格姆妇女医院 | 用于跨血脑屏障递送试剂的方法和组合物 |
-
2020
- 2020-11-20 PH PH1/2022/551247A patent/PH12022551247A1/en unknown
- 2020-11-20 JP JP2022529681A patent/JP7712271B2/ja active Active
- 2020-11-20 IL IL293140A patent/IL293140A/en unknown
- 2020-11-20 CN CN202080094086.2A patent/CN115023242B/zh active Active
- 2020-11-20 US US17/778,783 patent/US20230346981A1/en active Pending
- 2020-11-20 KR KR1020227021143A patent/KR20220116459A/ko active Pending
- 2020-11-20 CN CN202510215753.2A patent/CN119978146A/zh active Pending
- 2020-11-20 AU AU2020388404A patent/AU2020388404A1/en active Pending
- 2020-11-20 WO PCT/US2020/061464 patent/WO2021102234A1/en not_active Ceased
- 2020-11-20 BR BR112022009864A patent/BR112022009864A2/pt unknown
- 2020-11-20 CA CA3159113A patent/CA3159113A1/en active Pending
- 2020-11-20 MX MX2022006188A patent/MX2022006188A/es unknown
- 2020-11-20 EP EP20890214.8A patent/EP4061427A4/en active Pending
-
2022
- 2022-06-16 CO CONC2022/0008507A patent/CO2022008507A2/es unknown
-
2023
- 2023-10-20 US US18/491,365 patent/US20240100194A1/en active Pending
-
2025
- 2025-03-19 JP JP2025044678A patent/JP2025102825A/ja active Pending
- 2025-07-10 JP JP2025116303A patent/JP2025148448A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JPWO2021102234A5 (https=) | ||
| JP2021534809A (ja) | Aavカプシドの向性の再指向化 | |
| RU2017121593A (ru) | Векторы aav, нацеленные на центральную нервную систему | |
| US20020091094A1 (en) | Glutamic acid decarboxylase (GAD) based delivery system | |
| US20230104357A1 (en) | Combination of Retromer Pharmacological Chaperones and Exogenous Retromer for the Treatment of Alzheimer's Disease and Other Neurodegenerative Diseases and Disorders | |
| WO2022033454A1 (en) | Compositions and methods for dissolving protein aggregates | |
| JP2023542211A (ja) | 神経疾患を処置するための方法 | |
| US20200377887A1 (en) | Compositions and methods of treating huntington's disease | |
| JP7811415B2 (ja) | ビエッティ結晶性ジストロフィー治療のための遺伝子組換アデノ随伴ウイルスベクター | |
| WO2025038430A1 (en) | Aav capsid variants and uses thereof | |
| JP2020533968A (ja) | 組換えアデノ随伴ベクター | |
| JPWO2023154693A5 (https=) | ||
| WO2021032068A1 (zh) | Ptbp1抑制剂在预防和/或治疗功能性神经元死亡相关的神经系统疾病中的应用 | |
| TW202110486A (zh) | 使用糖苷水解酶改善基因療法載體向視網膜細胞的遞送 | |
| AU2025248711A1 (en) | Gene Therapy For Addiction Disorders | |
| US20230330267A1 (en) | Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain | |
| JPWO2018102665A5 (https=) | ||
| US20250025575A1 (en) | Compositions and Methods for Cell-Specific Expression of Target Genes | |
| US20240100132A1 (en) | Modulation of aav-based gene expression | |
| HK40095649A (zh) | 具有增强的转导效率并在脑中广泛分布的重组腺相关病毒载体的新型工程化衣壳血清型 | |
| JPWO2020242892A5 (https=) | ||
| WO2024154102A1 (en) | Compositions and methods for administration of therapeutic and diagnostic agents to the central nervous system | |
| HK40116026A (zh) | 脑梗死的非人灵长类动物模型的制作方法和脑梗死治疗用药物组合物 | |
| HK40114438A (zh) | Aav衣壳变体及其用途 | |
| RU2025119393A (ru) | Композиции и способы для лечения неврологических нарушений, связанных с недостаточностью бета-глюкозилцерамидазы 1 |