JPWO2021077000A5 - - Google Patents

Download PDF

Info

Publication number
JPWO2021077000A5
JPWO2021077000A5 JP2022522782A JP2022522782A JPWO2021077000A5 JP WO2021077000 A5 JPWO2021077000 A5 JP WO2021077000A5 JP 2022522782 A JP2022522782 A JP 2022522782A JP 2022522782 A JP2022522782 A JP 2022522782A JP WO2021077000 A5 JPWO2021077000 A5 JP WO2021077000A5
Authority
JP
Japan
Prior art keywords
disease
polypeptide
aav
vector system
cargo
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.)
Pending
Application number
JP2022522782A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022551986A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/056133 external-priority patent/WO2021077000A1/en
Publication of JP2022551986A publication Critical patent/JP2022551986A/ja
Publication of JPWO2021077000A5 publication Critical patent/JPWO2021077000A5/ja
Pending legal-status Critical Current

Links

JP2022522782A 2019-10-16 2020-10-16 修飾筋肉標的化組成物 Pending JP2022551986A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962916221P 2019-10-16 2019-10-16
US201962916207P 2019-10-16 2019-10-16
US62/916,221 2019-10-16
US62/916,207 2019-10-16
US202063018454P 2020-04-30 2020-04-30
US63/018,454 2020-04-30
US202063055252P 2020-07-22 2020-07-22
US63/055,252 2020-07-22
PCT/US2020/056133 WO2021077000A1 (en) 2019-10-16 2020-10-16 Engineered muscle targeting compositions

Publications (2)

Publication Number Publication Date
JP2022551986A JP2022551986A (ja) 2022-12-14
JPWO2021077000A5 true JPWO2021077000A5 (ko) 2023-10-18

Family

ID=75538392

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022522782A Pending JP2022551986A (ja) 2019-10-16 2020-10-16 修飾筋肉標的化組成物

Country Status (7)

Country Link
US (2) US20220340929A1 (ko)
EP (1) EP4045637A4 (ko)
JP (1) JP2022551986A (ko)
CN (1) CN115175991A (ko)
AU (1) AU2020368539A1 (ko)
CA (1) CA3153902A1 (ko)
WO (1) WO2021077000A1 (ko)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4244209A1 (en) 2020-11-11 2023-09-20 European Molecular Biology Laboratory Modified viral particles for gene therapy
US20240197918A1 (en) * 2021-04-23 2024-06-20 Locanabio, Inc. Tissue-targeted modified aav capsids and methods of use thereof
AR125406A1 (es) * 2021-04-23 2023-07-12 Univ Pennsylvania Nuevas composiciones con motivos selectivos para el cerebro y composiciones que las contienen
WO2022226375A1 (en) * 2021-04-23 2022-10-27 Locanabio, Inc. Tissue-targeted modified aav capsids and methods of use thereof
AU2022324116A1 (en) * 2021-08-05 2024-03-14 Massachusetts Institute Of Technology Engineered muscle targeting compositions
AU2022325165A1 (en) * 2021-08-05 2024-01-18 Insmed Incorporated Adeno-associated virus particles and methods of use thereof
AU2022334454A1 (en) * 2021-08-24 2024-03-14 Prime Medicine, Inc. Genome editing compositions and methods for treatment of retinopathy
EP4399316A1 (en) * 2021-09-08 2024-07-17 The Broad Institute, Inc. Engineered muscle and central nervous system compositions
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
AU2022360985A1 (en) * 2021-10-05 2024-05-16 Massachusetts Institute Of Technology Engineered cardiac muscle compositions
WO2023111351A1 (en) * 2021-12-17 2023-06-22 Christian Kupatt Aav vectors for gene therapy in endothelial cells
WO2023148617A1 (en) * 2022-02-01 2023-08-10 The Broad Institute, Inc. Adeno-associated viral vectors and uses thereof
WO2023183860A1 (en) * 2022-03-23 2023-09-28 University Of Delaware Engineered extracellular vesicles for targeted drug delivery to muscle
CN116870198B (zh) * 2023-09-06 2023-11-28 中国农业大学 一种调节骨骼肌损伤修复的方法

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002523105A (ja) 1998-08-27 2002-07-30 アバンテイス・フアルマ・エス・アー 異種遺伝子の運搬用の標的化アデノウイルスベクター
DE19933719A1 (de) 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
US7749492B2 (en) * 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
US20040142325A1 (en) * 2001-09-14 2004-07-22 Liat Mintz Methods and systems for annotating biomolecular sequences
WO2005075506A1 (en) * 2004-01-09 2005-08-18 The Scripps Research Institute Identification of endogenous trimerization domains in the adenovirus fiber protein that allow detargeting and retargeting of viral vectors
US8476418B2 (en) 2009-05-28 2013-07-02 Deutsches Krebsforschungszentrum Modified AAV capsid polypeptides
WO2015116568A2 (en) * 2014-01-28 2015-08-06 University Of Miami Muscle cell-targeting nanoparticles for vaccination and nucleic acid delivery, and methods of production and use thereof
WO2015191508A1 (en) * 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
CA2988486A1 (en) * 2015-06-10 2016-12-15 Association Institut De Myologie Combined therapy for duchenne muscular dystrophy
EP3384034B1 (en) 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
CA3097375A1 (en) * 2018-04-27 2019-10-31 Universitat Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
CN114729384A (zh) * 2019-09-12 2022-07-08 博德研究所 工程化腺相关病毒衣壳
CN114787179A (zh) * 2019-10-10 2022-07-22 坚固生物科技公司 经修饰的aav衣壳及其用途

Similar Documents

Publication Publication Date Title
JPWO2021077000A5 (ko)
JP6174318B2 (ja) アデノ随伴ウイルス構築物を含んでなるコンダクティング気道細胞を標的とするための組成物
ES2768763T3 (es) Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR
JP2020528734A5 (ko)
JP2021106619A5 (ko)
JP2021087431A5 (ko)
JP2020522269A5 (ko)
JP2019503649A5 (ko)
JPWO2020206189A5 (ko)
CN116096431A (zh) 肽修饰的aav衣壳
JP2020510447A5 (ko)
WO2020208032A1 (en) Hybrid promoters for muscle expression
JPWO2019152609A5 (ko)
Bulaklak et al. Therapeutic advances in musculoskeletal AAV targeting approaches
JPWO2020106916A5 (ko)
JP2020510433A5 (ko)
RU2019130004A (ru) Генная терапия при глазных заболеваниях
JPWO2021053222A5 (ko)
JPWO2019153009A5 (ko)
JPWO2020033473A5 (ko)
JPWO2019152474A5 (ko)
JPWO2020069339A5 (ko)
JPWO2021222831A5 (ko)
Flotte Adeno-associated virus-mediated gene transfer for lung diseases
JPWO2020214609A5 (ko)