KR20240122827A - 바이러스 벡터로 mybpc3 관련 비대형 심근병증을 치료하는 방법 및 조성물 - Google Patents

바이러스 벡터로 mybpc3 관련 비대형 심근병증을 치료하는 방법 및 조성물 Download PDF

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KR20240122827A
KR20240122827A KR1020247023075A KR20247023075A KR20240122827A KR 20240122827 A KR20240122827 A KR 20240122827A KR 1020247023075 A KR1020247023075 A KR 1020247023075A KR 20247023075 A KR20247023075 A KR 20247023075A KR 20240122827 A KR20240122827 A KR 20240122827A
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sequence
raav
nucleic acid
promoter
expression
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배리 존 번
페드로 크루즈
아이린 졸로투킨
와이들러 케이시
마누엘라 코르티
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유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드
애반티바이오, 인크.
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    • C07K14/4716—Muscle proteins, e.g. myosin, actin
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    • A61K38/1719—Muscle proteins, e.g. myosin or actin
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Immunology (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020247023075A 2021-12-10 2022-12-09 바이러스 벡터로 mybpc3 관련 비대형 심근병증을 치료하는 방법 및 조성물 Pending KR20240122827A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163288519P 2021-12-10 2021-12-10
US63/288,519 2021-12-10
PCT/US2022/081321 WO2023108157A1 (en) 2021-12-10 2022-12-09 Methods and compositions for treating mybpc3 related hypertrophic cardiomyopathy with a viral vector

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KR20240122827A true KR20240122827A (ko) 2024-08-13

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US (1) US20250041452A1 (https=)
EP (1) EP4444363A4 (https=)
JP (1) JP2024545186A (https=)
KR (1) KR20240122827A (https=)
AU (1) AU2022408189A1 (https=)
CA (1) CA3242262A1 (https=)
IL (1) IL313426A (https=)
WO (1) WO2023108157A1 (https=)

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Publication number Priority date Publication date Assignee Title
WO2025051970A2 (en) * 2023-09-06 2025-03-13 Dinaqor Ag Methods of treatment of hypertrophic cardiomyopathy with aav gene therapy vectors and therapeutic formulations
CN117959432A (zh) * 2023-12-13 2024-05-03 上海市第十人民医院 一种myl4基因在治疗或预防心房疾病中的应用
WO2025162449A1 (en) * 2024-01-30 2025-08-07 Inspirar Limited Compositions and methods for the treatment of heart disease

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Publication number Priority date Publication date Assignee Title
WO1999019471A1 (en) * 1997-10-16 1999-04-22 Board Of Regents, The University Of Texas System Models and treatments for cardiac hypertrophy in relation with nf-at3 function
WO2010037593A1 (en) * 2008-10-01 2010-04-08 Tet Systems Holding Gmbh & Co.Kg Tetracycline inducible transcription control sequence
EP3452101A2 (en) * 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
SG11202002276VA (en) * 2017-09-20 2020-04-29 4D Molecular Therapeutics Inc Adeno-associated virus variant capsids and methods of use thereof
CN109776680B (zh) * 2019-01-25 2020-05-12 浙江蓝盾药业有限公司 抗人pcsk9单克隆抗体及其用途
JP7785352B2 (ja) * 2019-08-14 2025-12-15 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 遺伝子治療のためのａａｖカプシドバリアント
WO2021154923A2 (en) * 2020-01-29 2021-08-05 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
WO2021163357A2 (en) * 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
MY209860A (en) * 2020-02-13 2025-08-07 Tenaya Therapeutics Inc Gene therapy vectors for treating heart disease

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EP4444363A1 (en) 2024-10-16
CA3242262A1 (en) 2023-06-15
JP2024545186A (ja) 2024-12-05
WO2023108157A1 (en) 2023-06-15
AU2022408189A1 (en) 2024-06-20
US20250041452A1 (en) 2025-02-06
EP4444363A4 (en) 2026-04-15
IL313426A (en) 2024-08-01

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