JPWO2021102234A5 - - Google Patents

Download PDF

Info

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
Application number
JP2022529681A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023503455A (ja
JP7712271B2 (ja
JP2023503455A5 (https=
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 JPWO2021102234A5 publication Critical patent/JPWO2021102234A5/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 (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)

* Cited by examiner, † Cited by third party
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)

* 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
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

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