EP-4707400-A3 - GENE THERAPIES FOR LYSOSOMAL DISORDERS
EP4707400A3EP 4707400 A3EP4707400 A3EP 4707400A3EP-4707400-A3
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.
Inventors
- ABELIOVICH, ASA
- HECKMAN, LAURA
- WONG, Li Chin
- LIN, Hsuan-Ni
- HEFTI, Franz
- RHINN, HERVE
Assignees
- Prevail Therapeutics, Inc.
Dates
- Publication Date
- 20260506
- Application Date
- 20200410