JP2023535808A5 - - Google Patents

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Publication number
JP2023535808A5
JP2023535808A5 JP2023506111A JP2023506111A JP2023535808A5 JP 2023535808 A5 JP2023535808 A5 JP 2023535808A5 JP 2023506111 A JP2023506111 A JP 2023506111A JP 2023506111 A JP2023506111 A JP 2023506111A JP 2023535808 A5 JP2023535808 A5 JP 2023535808A5
Authority
JP
Japan
Prior art keywords
polypeptide
gcase
seq
mutation
modified
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
JP2023506111A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023535808A (ja
Filing date
Publication date
Priority claimed from GBGB2011813.9A external-priority patent/GB202011813D0/en
Priority claimed from GBGB2100648.1A external-priority patent/GB202100648D0/en
Priority claimed from GBGB2105924.1A external-priority patent/GB202105924D0/en
Application filed filed Critical
Priority claimed from PCT/GB2021/051969 external-priority patent/WO2022023761A2/en
Publication of JP2023535808A publication Critical patent/JP2023535808A/ja
Publication of JP2023535808A5 publication Critical patent/JP2023535808A5/ja
Pending legal-status Critical Current

Links

JP2023506111A 2020-07-29 2021-07-29 安定性の改善された変異型ベータ-グルコセレブロシダーゼ Pending JP2023535808A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
GBGB2011813.9A GB202011813D0 (en) 2020-07-29 2020-07-29 Polypeptide
GB2011813.9 2020-07-29
GBGB2100648.1A GB202100648D0 (en) 2021-01-18 2021-01-18 Polypeptide
GB2100648.1 2021-01-18
GB2105924.1 2021-04-26
GBGB2105924.1A GB202105924D0 (en) 2021-04-26 2021-04-26 Polypeptide
PCT/GB2021/051969 WO2022023761A2 (en) 2020-07-29 2021-07-29 Polypeptide

Publications (2)

Publication Number Publication Date
JP2023535808A JP2023535808A (ja) 2023-08-21
JP2023535808A5 true JP2023535808A5 (https=) 2024-08-07

Family

ID=77265101

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023506111A Pending JP2023535808A (ja) 2020-07-29 2021-07-29 安定性の改善された変異型ベータ-グルコセレブロシダーゼ

Country Status (16)

Country Link
US (1) US20240261433A1 (https=)
EP (2) EP4118200B1 (https=)
JP (1) JP2023535808A (https=)
KR (1) KR20230042513A (https=)
CN (1) CN116322744A (https=)
AU (1) AU2021316875B2 (https=)
BR (1) BR112023001583A2 (https=)
CA (1) CA3189801A1 (https=)
DK (1) DK4118200T3 (https=)
ES (1) ES3057448T3 (https=)
FI (1) FI4118200T3 (https=)
HR (1) HRP20251591T1 (https=)
IL (1) IL299811A (https=)
MX (1) MX2023001137A (https=)
PT (1) PT4118200T (https=)
WO (1) WO2022023761A2 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202532096A (zh) 2023-12-08 2025-08-16 美商戴納立製藥公司 經修飾之葡萄糖腦苷脂酶多肽及其使用方法
WO2025166045A1 (en) 2024-01-31 2025-08-07 Alector Llc β-GLUCOCEREBROSIDASE ENZYMES, FUSION PROTEINS AND COMPLEXES COMPRISING THE SAME, AND METHODS OF USE THEREOF
CN118086341B (zh) * 2024-04-25 2024-08-02 上海凌医生物科技有限公司 在肝脏中高表达人源葡萄糖脑苷脂酶基因的表达框
WO2026006173A1 (en) 2024-06-24 2026-01-02 Denali Therapeutics Inc. Fusion proteins comprising modified glucocerebrosidase polypeptides and methods thereof

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6582692B1 (en) * 1999-11-17 2003-06-24 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
AU2352201A (en) * 1999-12-30 2001-07-16 Maxygen Aps Improved lysosomal enzymes and lysosomal enzyme activators
EP2154969B1 (en) 2007-05-16 2015-11-18 The Brigham and Women's Hospital, Inc. Treatment of synucleinopathies
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
SI2638152T1 (sl) * 2010-11-08 2016-12-30 Amicus Therapeutics, Inc. Variantni in rekombinantni proteini beta-glukocerebrozidaze s povišano stabilnostjo in povišano ohranjeno katalitično aktivnostjo
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US9574184B2 (en) * 2013-09-25 2017-02-21 Children's Hospital Medical Center Lysosomal protein targeting sequence and therapeutic applications of same
MX2018012537A (es) * 2016-04-15 2019-02-25 Univ Pennsylvania Terapia de genes para tratar hemofilia a.
AU2018346104B2 (en) 2017-10-03 2023-07-27 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
BR112020006661A2 (pt) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
WO2020012149A1 (en) 2018-07-13 2020-01-16 Ucl Business Ltd Glucocerebrosidase gene therapy
US20220125892A1 (en) * 2019-02-01 2022-04-28 OXYRANE UK Ltd. Glucocerebrosidase polypeptides

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