KR20230016175A - Angptl3의 염기 편집 및 질환 치료를 위한 이의 사용 방법 - Google Patents

Angptl3의 염기 편집 및 질환 치료를 위한 이의 사용 방법 Download PDF

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KR20230016175A
KR20230016175A KR1020227038926A KR20227038926A KR20230016175A KR 20230016175 A KR20230016175 A KR 20230016175A KR 1020227038926 A KR1020227038926 A KR 1020227038926A KR 20227038926 A KR20227038926 A KR 20227038926A KR 20230016175 A KR20230016175 A KR 20230016175A
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composition
editing
guide rna
administered
angptl3
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알렉산드라 체드윅
칼란토타틸 지 라지브
앨런 로드
크리스토퍼 쳉
캐롤라인 레이스
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버브 테라퓨틱스, 인크.
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    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/6454—Dibasic site splicing serine proteases, e.g. kexin (3.4.21.61); furin (3.4.21.75) and other proprotein convertases
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    • C07K2319/00—Fusion polypeptide
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KR1020227038926A 2020-04-09 2021-04-09 Angptl3의 염기 편집 및 질환 치료를 위한 이의 사용 방법 Pending KR20230016175A (ko)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202063007803P 2020-04-09 2020-04-09
US202063007797P 2020-04-09 2020-04-09
US63/007,803 2020-04-09
US63/007,797 2020-04-09
US202063045033P 2020-06-26 2020-06-26
US202063045032P 2020-06-26 2020-06-26
US63/045,033 2020-06-26
US63/045,032 2020-06-26
US202163136087P 2021-01-11 2021-01-11
US63/136,087 2021-01-11
PCT/US2021/026729 WO2021207710A2 (en) 2020-04-09 2021-04-09 Base editing of angptl3 and methods of using same for treatment of disease

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KR20230016175A true KR20230016175A (ko) 2023-02-01

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Application Number Title Priority Date Filing Date
KR1020227038926A Pending KR20230016175A (ko) 2020-04-09 2021-04-09 Angptl3의 염기 편집 및 질환 치료를 위한 이의 사용 방법
KR1020227038947A Pending KR20230016176A (ko) 2020-04-09 2021-04-09 Pcsk9의 염기 편집 및 질환 치료를 위한 이의 사용 방법

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KR1020227038947A Pending KR20230016176A (ko) 2020-04-09 2021-04-09 Pcsk9의 염기 편집 및 질환 치료를 위한 이의 사용 방법

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US (5) US12029795B2 (https=)
EP (3) EP4133072A4 (https=)
JP (4) JP7457833B2 (https=)
KR (2) KR20230016175A (https=)
CN (2) CN116096429A (https=)
AU (4) AU2021252991B2 (https=)
BR (2) BR112022020407A2 (https=)
CA (2) CA3179863A1 (https=)
GB (8) GB2613457B (https=)
IL (2) IL297193A (https=)
MX (1) MX2022012683A (https=)
PH (2) PH12022553015A1 (https=)
WO (3) WO2021207710A2 (https=)

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EP3585895A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Compositions and methods for gene editing
MX2022012683A (es) * 2020-04-09 2023-01-11 Verve Therapeutics Inc Edicion de bases de pcsk9 y metodos de uso de la misma para el tratamiento de enfermedades.
WO2022115645A1 (en) 2020-11-25 2022-06-02 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids, and related methods of use
CA3235827A1 (en) * 2021-10-21 2023-04-27 Wei Hsi Yeh Genome editing compositions and methods for treatment of retinitis pigmentosa
WO2023166425A1 (en) * 2022-03-01 2023-09-07 Crispr Therapeutics Ag Methods and compositions for treating angiopoietin-like 3 (angptl3) related conditions
US20230293646A1 (en) * 2022-03-21 2023-09-21 Crispr Therapeutics Ag Methods and compositions for treating lipoprotein-related diseases
CA3256897A1 (en) 2022-05-25 2023-11-30 Akagera Medicines, Inc. Lipid nanoparticles for the administration of nucleic acids and their methods of use
GB2634837A (en) 2022-06-07 2025-04-23 Scribe Therapeutics Inc Compositions and methods for the targeting of PCSK9
WO2024061296A2 (en) * 2022-09-22 2024-03-28 Accuredit Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treatment of hypercholesterolemia and/or cardiovascular disease
KR20250124819A (ko) * 2022-12-21 2025-08-20 인텔리아 테라퓨틱스, 인크. 프로단백질 전환효소 서브틸리신 켁신 9(pcsk9) 편집을 위한 조성물 및 방법
WO2024152058A1 (en) * 2023-01-13 2024-07-18 The Trustees Of The University Of Pennsylvania Compositions and methods for the treatment of hereditary tyrosinemia type 1
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