Invention Grant
- Patent Title: Gene therapies for lysosomal disorders
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Application No.: US16846065Application Date: 2020-04-10
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Publication No.: US11999974B2Publication Date: 2024-06-04
- Inventor: Asa Abeliovich , Laura Heckman , Herve Rhinn , Li Chin Wong , Hsuan-Ni Lin , Franz Hefti
- Applicant: Prevail Therapeutics, Inc.
- Applicant Address: US NY New York
- Assignee: Prevail Therapeutics, Inc.
- Current Assignee: Prevail Therapeutics, Inc.
- Current Assignee Address: US NY New York
- Agency: COOLEY LLP
- Agent Ivor Elrifi
- Main IPC: C12N7/00
- IPC: C12N7/00 ; A61K48/00 ; A61P25/28 ; C07K14/435 ; C12N9/02 ; G01N33/68

Abstract:
The disclosure relates to compositions and methods for treatment of diseases associated with aberrant lysosomal function, such as fronto-temporal dementia (FTD). The disclosure also provides expression constructs comprising a transgene encoding progranulin or a portion thereof. The disclosure provides methods of treating FTD by administering such expression constructs to a subject in need thereof.
Public/Granted literature
- US20200332265A1 GENE THERAPIES FOR LYSOSOMAL DISORDERS Public/Granted day:2020-10-22
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