JPWO2021102107A5 - - Google Patents

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Publication number
JPWO2021102107A5
JPWO2021102107A5 JP2022529007A JP2022529007A JPWO2021102107A5 JP WO2021102107 A5 JPWO2021102107 A5 JP WO2021102107A5 JP 2022529007 A JP2022529007 A JP 2022529007A JP 2022529007 A JP2022529007 A JP 2022529007A JP WO2021102107 A5 JPWO2021102107 A5 JP WO2021102107A5
Authority
JP
Japan
Prior art keywords
seq
nucleic acid
sequence
aav vector
gaa
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
JP2022529007A
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English (en)
Japanese (ja)
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JP2023503046A5 (https=
JP2023503046A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/061223 external-priority patent/WO2021102107A1/en
Publication of JP2023503046A publication Critical patent/JP2023503046A/ja
Publication of JP2023503046A5 publication Critical patent/JP2023503046A5/ja
Publication of JPWO2021102107A5 publication Critical patent/JPWO2021102107A5/ja
Pending legal-status Critical Current

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JP2022529007A 2019-11-19 2020-11-19 ポンペ病およびリソソーム障害を処置するための肝臓特異的プロモーターを含む治療用アデノ随伴ウイルス Pending JP2023503046A (ja)

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

Publications (3)

Publication Number Publication Date
JP2023503046A JP2023503046A (ja) 2023-01-26
JP2023503046A5 JP2023503046A5 (https=) 2023-11-28
JPWO2021102107A5 true JPWO2021102107A5 (https=) 2023-11-28

Family

ID=75981066

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022529007A Pending JP2023503046A (ja) 2019-11-19 2020-11-19 ポンペ病およびリソソーム障害を処置するための肝臓特異的プロモーターを含む治療用アデノ随伴ウイルス

Country Status (11)

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=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
KR102814185B1 (ko) * 2023-07-20 2025-06-12 제닉스큐어 주식회사 라포라병을 예방 또는 치료하기 위한 재조합 아데노 연관 바이러스 및 이를 포함하는 약학적 조성물
AU2024317483A1 (en) * 2023-07-28 2026-01-29 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2001266740A1 (en) * 2000-06-06 2001-12-17 Genelabs Technologies, Inc. Promoters for regulated gene expression
ATE521701T1 (de) * 2003-01-22 2011-09-15 Univ Duke Verbesserte konstrukte zur expression lysosomaler polypeptide
US7521211B2 (en) * 2005-04-05 2009-04-21 Regeneron Pharmaceuticals, Inc IGF-1 and IGF-2 chimeric polypeptides and therapeutic uses thereof
EP2162540A2 (en) * 2007-05-22 2010-03-17 Amgen Inc. Compositions and methods for producing bioactive fusion proteins
HRP20170698T1 (hr) * 2008-05-07 2017-09-22 Biomarin Pharmaceutical Inc. Lizosomski usmjereni peptidi i njihove uporabe
AU2010366066B2 (en) * 2010-12-22 2016-01-14 Fondazione Telethon Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses
MX2013012345A (es) * 2011-04-22 2015-05-07 Genzyme Corp Alfa glucosidasa acida modificada con procesamiento acelerado.
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
EP3293260A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3293259A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
US12296026B2 (en) * 2018-02-05 2025-05-13 Astellas Gene Therapies, Inc. Transcription regulatory elements and uses thereof
JP7779653B2 (ja) * 2018-02-07 2025-12-03 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 治療用タンパク質送達のための方法および組成物
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

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