Invention Application
- Patent Title: VARIANTS OF BETA-GLUCOCEREBROSIDASE FOR USE IN TREATING GAUCHER DISEASE
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Application No.: US17915500Application Date: 2021-03-29
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Publication No.: US20230132790A1Publication Date: 2023-05-04
- Inventor: Anthony H. FUTERMAN , Israel SILMAN , Joel L. SUSSMAN , Sarel FLEISHMAN , Adi GOLDENZWEIG , Sarka POKORNA , Yaacov ASHANI , Olga KHERSONSKY
- Applicant: Yeda Research and Development Co. Ltd.
- Applicant Address: IL Rehovot
- Assignee: Yeda Research and Development Co. Ltd.
- Current Assignee: Yeda Research and Development Co. Ltd.
- Current Assignee Address: IL Rehovot
- Priority: IL273684 20200329
- International Application: PCT/IL2021/050357 WO 20210329
- Main IPC: C12N9/24
- IPC: C12N9/24
Abstract:
A genetically modified human beta-glucocerebrosidase (GCase) is disclosed. The genetically modified GCase comprising an amino acid sequence at least 85% identical to SEQ ID NO: 2; and comprising mutations at coordinates L34P, K224N/G, T369E and N370D, where the coordinates correspond to said SEQ ID NO: 2; and capable of catalyzing hydrolysis of a glycolipid glucosylceramide (GlcCer). Pharmaceutical compositions comprising the genetically modified GCase and therapeutic methods of using same are also disclosed.
Information query
