JP2023534999A5 - - Google Patents

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Publication number
JP2023534999A5
JP2023534999A5 JP2023504171A JP2023504171A JP2023534999A5 JP 2023534999 A5 JP2023534999 A5 JP 2023534999A5 JP 2023504171 A JP2023504171 A JP 2023504171A JP 2023504171 A JP2023504171 A JP 2023504171A JP 2023534999 A5 JP2023534999 A5 JP 2023534999A5
Authority
JP
Japan
Prior art keywords
polypeptide
aav
mutation
viral
disease
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
JP2023504171A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023534999A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/042812 external-priority patent/WO2022020616A1/en
Publication of JP2023534999A publication Critical patent/JP2023534999A/ja
Publication of JP2023534999A5 publication Critical patent/JP2023534999A5/ja
Pending legal-status Critical Current

Links

JP2023504171A 2020-07-22 2021-07-22 操作された筋肉標的化組成物 Pending JP2023534999A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063055265P 2020-07-22 2020-07-22
US63/055,265 2020-07-22
US202063107394P 2020-10-29 2020-10-29
US63/107,394 2020-10-29
US202163183038P 2021-05-02 2021-05-02
US63/183,038 2021-05-02
PCT/US2021/042812 WO2022020616A1 (en) 2020-07-22 2021-07-22 Engineered muscle targeting compositions

Publications (2)

Publication Number Publication Date
JP2023534999A JP2023534999A (ja) 2023-08-15
JP2023534999A5 true JP2023534999A5 (https=) 2024-07-26

Family

ID=79728337

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023504171A Pending JP2023534999A (ja) 2020-07-22 2021-07-22 操作された筋肉標的化組成物

Country Status (13)

Country Link
EP (1) EP4185702A4 (https=)
JP (1) JP2023534999A (https=)
KR (1) KR20230053591A (https=)
CN (1) CN116194153A (https=)
AU (1) AU2021314256A1 (https=)
BR (1) BR112023000909A2 (https=)
CA (1) CA3183530A1 (https=)
CL (2) CL2023000204A1 (https=)
CO (1) CO2023000866A2 (https=)
IL (1) IL299431A (https=)
MX (1) MX2023000998A (https=)
TW (1) TW202217003A (https=)
WO (1) WO2022020616A1 (https=)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12552838B2 (en) 2020-04-20 2026-02-17 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
US20240115734A1 (en) * 2021-02-09 2024-04-11 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
CA3216419A1 (en) * 2021-04-23 2022-10-27 Greg NACHTRAB Tissue-targeted modified aav capsids and methods of use thereof
JP2024530023A (ja) * 2021-08-05 2024-08-14 ザ・ブロード・インスティテュート・インコーポレイテッド 操作された筋肉標的化組成物
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
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
EP4504952A4 (en) * 2022-04-07 2026-04-08 Kate Therapeutics Inc COMPOSITIONS AND METHODS FOR MUSCULAR DISORDERS
WO2023198828A1 (en) * 2022-04-13 2023-10-19 Universitat Autònoma De Barcelona Treatment of neuromuscular diseases via gene therapy that expresses klotho protein
ES3057171T3 (en) * 2022-06-10 2026-02-26 Genethon Peptide-modified aav capsid with enhanced muscle transduction efficiency
EP4634394A2 (en) * 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
EP4680741A2 (en) 2023-03-15 2026-01-21 RegenxBio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025090858A1 (en) * 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
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
US20250276095A1 (en) 2024-03-04 2025-09-04 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
WO2025250909A1 (en) 2024-05-31 2025-12-04 Sarepta Therapeutics, Inc. Muscle-tropic recombinant aav
CN118667855A (zh) * 2024-05-31 2024-09-20 扬州大学 一种具有肌肉靶向性促进骨形成的噬菌体的构建方法及其应用
WO2026006341A1 (en) 2024-06-24 2026-01-02 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy
WO2026030244A1 (en) 2024-07-30 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of limb girdle muscular dystrophy 2a
WO2026041758A1 (en) 2024-08-22 2026-02-26 Genethon Modified aav capsid with enhanced transduction efficiency
WO2026050402A1 (en) 2024-08-29 2026-03-05 Kate Therapeutics, Inc. Liver de-targeted muscle tropic capsids
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064739A1 (en) * 2024-09-23 2026-03-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy
WO2026069251A1 (en) * 2024-09-30 2026-04-02 Sarepta Therapeutics, Inc. Adeno-associated virus capsid antibodies and fragments thereof

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210363193A1 (en) * 2018-04-27 2021-11-25 Universität Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
US12397084B2 (en) * 2018-07-23 2025-08-26 Georgia Tech Research Corporation Synthetic hydrogel carriers for muscle repair
EP4028064A1 (en) * 2019-09-12 2022-07-20 The Broad Institute, Inc. Engineered adeno-associated virus capsids

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