JP2023503455A5 - - Google Patents

Info

Publication number
JP2023503455A5
JP2023503455A5 JP2022529681A JP2022529681A JP2023503455A5 JP 2023503455 A5 JP2023503455 A5 JP 2023503455A5 JP 2022529681 A JP2022529681 A JP 2022529681A JP 2022529681 A JP2022529681 A JP 2022529681A JP 2023503455 A5 JP2023503455 A5 JP 2023503455A5
Authority
JP
Japan
Prior art keywords
capsid protein
modified
seq
targeting peptide
different brain
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
Application number
JP2022529681A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023503455A (ja
JP7712271B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/061464 external-priority patent/WO2021102234A1/en
Publication of JP2023503455A publication Critical patent/JP2023503455A/ja
Publication of JP2023503455A5 publication Critical patent/JP2023503455A5/ja
Priority to JP2025044678A priority Critical patent/JP2025102825A/ja
Priority to JP2025116303A priority patent/JP2025148448A/ja
Application granted granted Critical
Publication of JP7712271B2 publication Critical patent/JP7712271B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2022529681A 2019-11-22 2020-11-20 アデノ随伴ウイルスベクター変種 Active JP7712271B2 (ja)

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 (3)

Publication Number Publication Date
JP2023503455A JP2023503455A (ja) 2023-01-30
JP2023503455A5 true 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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4136457B1 (en) * 2020-04-17 2025-02-19 Regeneron Pharmaceuticals, Inc. Detection assays for coronavirus neutralizing antibodies
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
JP2025514794A (ja) * 2022-04-22 2025-05-09 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア ヒト上衣特異的プロモーターおよびその使用方法
CN119654334A (zh) * 2022-05-11 2025-03-18 费城儿童医院 用于靶向深层脑结构的腺相关病毒载体
AU2023267671A1 (en) * 2022-05-13 2024-12-05 The Children's Hospital Of Philadelphia Adeno-associated viral vectors for targeting brain microvasculature
JP2025521111A (ja) 2022-05-16 2025-07-08 ジェンザイム・コーポレーション 異染性白質ジストロフィーを治療する方法
WO2024017387A1 (en) 2022-07-22 2024-01-25 Shanghai Vitalgen Biopharma Co., Ltd. Novel aav capsids for targeting nervous system and uses thereof
KR102870168B1 (ko) * 2022-08-26 2025-10-13 김선아 활막 표적화 화합물 및 이의 용도
IL319666A (en) * 2022-09-19 2025-05-01 Childrens Hospital Philadelphia AV evolution at single-cell resolution using SPLIT-SEQ
IL322459A (en) * 2023-01-31 2025-09-01 Childrens Hospital Philadelphia Innovative gene therapy construct for cln2 disease
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN116970041B (zh) * 2023-07-21 2024-02-09 广州派真生物技术有限公司 腺相关病毒突变体及其应用
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025090598A1 (en) * 2023-10-23 2025-05-01 The Children's Hospital Of Philadelphia Aav vectors for treatment of cln2 disease
WO2025090631A1 (en) * 2023-10-23 2025-05-01 The Children's Hospital Of Philadelphia Aav vectors for delivery of apoe2
US20250152747A1 (en) * 2023-11-15 2025-05-15 Genzyme Corporation Methods of treating gaucher disease and gba-parkinson’s disease
US20250152744A1 (en) * 2023-11-15 2025-05-15 Genzyme Corporation Methods of treating neurodegenerative disorders
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
CN121265801A (zh) * 2025-06-06 2026-01-06 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)

* Cited by examiner, † Cited by third party
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
PT1453547T (pt) * 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
ES2739288T3 (es) * 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
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
US11905312B2 (en) * 2017-02-15 2024-02-20 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
RU2019140862A (ru) * 2017-05-12 2021-06-15 Дзе Чилдрен'З Хоспитал Оф Филадельфия Варианты сульфамидазы (sgsh), векторы, композиции и способы, и применения для лечения мукополисахаридоз типа iiia (mps iiia)
RU2770922C2 (ru) * 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения
AU2018338728B2 (en) * 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) 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
WO2019222329A1 (en) * 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2020014471A1 (en) * 2018-07-11 2020-01-16 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of agents across the blood-brain barrier

Similar Documents

Publication Publication Date Title
JP2023503455A5 (https=)
JP2021534809A (ja) Aavカプシドの向性の再指向化
WO2023235791A1 (en) Aav capsid variants and uses thereof
KR20190117571A (ko) 혈관계를 통한 유전자 전달 방법 및 조성물
JP2022066336A5 (https=)
JP2020500526A5 (https=)
AU2021325233B2 (en) Compositions and methods for dissolving protein aggregates
JP2022545226A5 (https=)
JP2023535576A5 (https=)
WO2021031810A1 (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
JP2021525245A5 (https=)
TW202444917A (zh) Aav衣殼變異體及其用途
JP2020533968A (ja) 組換えアデノ随伴ベクター
JP2022533451A5 (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
JP2025516136A (ja) 神経変性疾患の治療のための組み換えaavベクター
JP2024515902A5 (https=)
Bouju et al. Molecular cloning and functional expression of a novel human gene encoding two 41–43 kDa skeletal muscle internal membrane proteins
CN117916365A (zh) 用于治疗结晶样视网膜变性的重组腺相关病毒载体
JP2024521401A5 (https=)
JP2023538130A5 (https=)
JP2024505197A5 (https=)
JP2025508429A5 (https=)
JP2023544141A (ja) AIMP2-DX2および必要に応じてmiR-142の標的配列ならびにその組成物を使用した神経系疾患を処置する方法
JP2023505271A5 (https=)