WO2025181155 - HUMAN BETA-GLUCOCEREBROSIDASE BINDERS AND USES THEREOF

National phase entry is expected:
Publication Number WO/2025/181155
Publication Date 04.09.2025
International Application No. PCT/EP2025/055198
International Filing Date 26.02.2025
Title **
[English] HUMAN BETA-GLUCOCEREBROSIDASE BINDERS AND USES THEREOF
[French] LIANTS DE BÊTA-GLUCOCÉRÉBROSIDASE HUMAINE ET LEURS UTILISATIONS
Applicants **
VIB VZW
VRIJE UNIVERSITEIT BRUSSEL
UNIVERSITÀ DEGLI STUDI DI PADOVA
Inventors
VERSÉES, Wim
DAL MASO, Thomas
PLOTEGHER, Nicoletta
GREGGIO, Elisa
SINISGALLI, Chiara
Priority Data
24159588.3   26.02.2024   EP
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Quotation for National Phase entry

Country StagesTotal
China Filing, Examination, Granting2770
EPO Filing, Examination, Granting11150
Japan Filing, Examination, Granting2174
South Korea Filing, Examination, Granting2050
USA Filing, Examination, Granting6990
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Total: 25,134
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Abstract[English] This application relates to polypeptide agents and compositions specifically binding human beta-glucocerebrosidase (GCase, GBA1). Particularly, immunoglobulin single variable domains (ISVDs) are described which allosterically bind human GCase, thereby positively modulating GCase in its stability and/or catalytic activity. The invention relates further to vectors and nucleic acids encoding such ISVD-based modulators. Also encompassed are compositions, in particular pharmaceutical compositions, containing such ISVD modulators. The GCase-specific allosteric modulators, specifically the ISVDs and compositions described herein, may be used for prevention and/or treatment of GCase-related diseases, including Gaucher disease (GD) and Parkinson's disease (PD).[French] La présente invention concerne des agents polypeptidiques et des compositions se liant spécifiquement à la bêta-glucocérébrosidase humaine (GCase, GBA1). En particulier, l'invention concerne des domaines variables uniques d'immunoglobuline (ISVD) qui se lient de manière allostérique à la GCase humaine, ce qui permet de moduler positivement la GCase au niveau de sa stabilité et/ou de son activité catalytique. L'invention concerne en outre des vecteurs et des acides nucléiques codant pour de tels modulateurs à base d'ISVD. L'invention concerne également des compositions, en particulier des compositions pharmaceutiques, contenant de tels modulateurs ISVD. Les modulateurs allostériques spécifiques de la GCase, en particulier les ISVD et les compositions décrites ici, peuvent être utilisés pour la prévention et/ou le traitement de maladies associées à la GCase, notamment la maladie de Gaucher (MG) et la maladie de Parkinson (MP).

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