KR20220098384A - 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스 - Google Patents

폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스 Download PDF

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KR20220098384A
KR20220098384A KR1020227020169A KR20227020169A KR20220098384A KR 20220098384 A KR20220098384 A KR 20220098384A KR 1020227020169 A KR1020227020169 A KR 1020227020169A KR 20227020169 A KR20227020169 A KR 20227020169A KR 20220098384 A KR20220098384 A KR 20220098384A
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seq
nucleic acid
sequence
gaa
aav vector
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마이클 로버츠
후안 마누엘 이글레시아스
안나 트레티아코바
마이클 더블유. 오캘러건
아킬레 프랑수아
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아스클레피오스 바이오파마슈티컬, 인크.
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    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
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    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • C—CHEMISTRY; METALLURGY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
    • C12N2810/85—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)

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KR1020227020169A 2019-11-19 2020-11-19 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스 Pending KR20220098384A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962937556P 2019-11-19 2019-11-19
US201962937583P 2019-11-19 2019-11-19
US62/937,556 2019-11-19
US62/937,583 2019-11-19
US202063023570P 2020-05-12 2020-05-12
US63/023,570 2020-05-12
PCT/US2020/061223 WO2021102107A1 (en) 2019-11-19 2020-11-19 Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders

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Publication Number Publication Date
KR20220098384A true KR20220098384A (ko) 2022-07-12

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KR1020227020169A Pending KR20220098384A (ko) 2019-11-19 2020-11-19 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스

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Country Link
US (1) US20230038520A1 (https=)
EP (1) EP4061946A4 (https=)
JP (1) JP2023503046A (https=)
KR (1) KR20220098384A (https=)
CN (1) CN116096895A (https=)
AU (1) AU2020388634A1 (https=)
CA (1) CA3159018A1 (https=)
IL (1) IL293068A (https=)
MX (1) MX2022005916A (https=)
PH (1) PH12022551229A1 (https=)
WO (1) WO2021102107A1 (https=)

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KR20240159745A (ko) 2023-04-28 2024-11-06 아주대학교산학협력단 유산균 유래 nadh 산화효소를 이용한 비알코올성 지방간염의 유전자 치료제
WO2025018822A1 (ko) * 2023-07-20 2025-01-23 제닉스큐어 주식회사 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물
KR20250018363A (ko) * 2023-07-20 2025-02-05 제닉스큐어 주식회사 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물

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WO2020102645A1 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease
US20240287148A1 (en) * 2021-07-15 2024-08-29 Cornell University Engineered biomolecules for nutrient reprogramming
KR20240073025A (ko) * 2021-08-25 2024-05-24 캔브리지 파마슈티컬스, 인크. 간-향성 캡시드 단백질 및 알파-갈락토시다제를 포함하는 aav 입자 및 파브리 질환을 치료하기 위한 이의 용도
TW202338095A (zh) * 2021-08-25 2023-10-01 美商北海康成製藥有限公司 包含肝臟向性衣殼蛋白質及酸α－葡萄糖苷酶（ＧＡＡ）之ＡＡＶ顆粒及其用於治療龐貝氏病（ＰＯＭＰＥ ＤＩＳＥＡＳＥ）之用途
MX2024009003A (es) * 2022-01-21 2024-09-17 Astrazeneca Ireland Ltd Terapia genica para la enfermedad de gaucher.
KR20240145491A (ko) * 2022-02-03 2024-10-07 아스텔라스 진 테라피스, 인크. 폼페병의 개선된 치료를 위한 조성물 및 방법
WO2023164060A2 (en) * 2022-02-25 2023-08-31 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease with long term cessation of gaa enzyme replacement therapy
WO2023172975A1 (en) * 2022-03-08 2023-09-14 University Of Massachusetts Methods of raav packaging
EP4493704A2 (en) * 2022-03-18 2025-01-22 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus using codon optimized nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with signal peptide modifications
WO2024055272A1 (zh) * 2022-09-16 2024-03-21 复旦大学附属中山医院 能高效表达目的基因的mRNA载体系统、其构建及应用
WO2024212961A1 (en) * 2023-04-10 2024-10-17 Skyline Therapeutics (Shanghai) Co., Ltd. Recombinant aav for the gene therapy of pompe disease
EP4698655A1 (en) * 2023-04-20 2026-02-25 AskBio Inc. Liver-specific regulatory nucleic acid sequences
AU2024317483A1 (en) * 2023-07-28 2026-01-29 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease

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KR20210005154A (ko) * 2018-04-30 2021-01-13 아미쿠스 세라퓨틱스, 인코포레이티드 유전자 요법 작제물 및 사용 방법
WO2020102645A1 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240159745A (ko) 2023-04-28 2024-11-06 아주대학교산학협력단 유산균 유래 nadh 산화효소를 이용한 비알코올성 지방간염의 유전자 치료제
WO2025018822A1 (ko) * 2023-07-20 2025-01-23 제닉스큐어 주식회사 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물
KR20250018363A (ko) * 2023-07-20 2025-02-05 제닉스큐어 주식회사 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물

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US20230038520A1 (en) 2023-02-09
IL293068A (en) 2022-07-01
TW202132570A (zh) 2021-09-01
WO2021102107A1 (en) 2021-05-27
EP4061946A4 (en) 2024-03-06
CA3159018A1 (en) 2021-05-27
CN116096895A (zh) 2023-05-09
MX2022005916A (es) 2022-08-04
JP2023503046A (ja) 2023-01-26
PH12022551229A1 (en) 2023-07-31
AU2020388634A1 (en) 2022-06-23
EP4061946A1 (en) 2022-09-28

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