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
Other languages
English (en)
Japanese (ja)
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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 JPWO2021102107A5 publication Critical patent/JPWO2021102107A5/ja
Pending legal-status Critical Current

Links

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
US62/937,583 2019-11-19
US62/937,556 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 (2)

Publication Number Publication Date
JP2023503046A JP2023503046A (ja) 2023-01-26
JPWO2021102107A5 true JPWO2021102107A5 (zh) 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 (zh)
EP (1) EP4061946A4 (zh)
JP (1) JP2023503046A (zh)
KR (1) KR20220098384A (zh)
CN (1) CN116096895A (zh)
AU (1) AU2020388634A1 (zh)
CA (1) CA3159018A1 (zh)
IL (1) IL293068A (zh)
MX (1) MX2022005916A (zh)
TW (1) TW202132570A (zh)
WO (1) WO2021102107A1 (zh)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3655041A4 (en) * 2017-08-25 2021-04-21 Ovid Therapeutics Inc RECOMBINANT ADENO-ASSOCIATED VECTORS
JP2022513067A (ja) * 2018-11-16 2022-02-07 アスクレピオス バイオファーマシューティカル, インコーポレイテッド ポンペ病を処置するための治療用アデノ随伴ウイルス
US20240287148A1 (en) * 2021-07-15 2024-08-29 Cornell University Engineered biomolecules for nutrient reprogramming
MX2024009003A (es) * 2022-01-21 2024-09-17 Astrazeneca Ireland Ltd Terapia genica para la enfermedad de gaucher.
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
WO2023177885A2 (en) * 2022-03-18 2023-09-21 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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040248262A1 (en) * 2003-01-22 2004-12-09 Koeberl Dwight D. Constructs for expressing lysomal polypeptides
US7521211B2 (en) * 2005-04-05 2009-04-21 Regeneron Pharmaceuticals, Inc IGF-1 and IGF-2 chimeric polypeptides and therapeutic uses thereof
JP5591691B2 (ja) * 2007-05-22 2014-09-17 アムジエン・インコーポレーテツド 生物活性を有する融合タンパク質を作製するための組成物及び方法
JP2014513952A (ja) * 2011-04-22 2014-06-19 ジェンザイム・コーポレーション 促進されたプロセシングを備えた修飾された酸性アルファグルコシダーゼ
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
MA51753A (fr) * 2018-02-05 2020-12-16 Audentes Therapeutics Inc Éléments de régulation de la transcription et utilisations associées
CN112040985A (zh) * 2018-02-07 2020-12-04 瑞泽恩制药公司 用于递送治疗性蛋白质的方法和组合物
CN112513071A (zh) * 2018-04-30 2021-03-16 阿米库斯治疗学公司 基因治疗构建体和使用方法
JP2022513067A (ja) * 2018-11-16 2022-02-07 アスクレピオス バイオファーマシューティカル, インコーポレイテッド ポンペ病を処置するための治療用アデノ随伴ウイルス

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