Compositions and methods for delivering syntaxin-binding protein-1
TW202635892APending Publication Date: 2026-09-01NEUROCRINE BIOSCIENCES INC +1
View PDF 0 Cites 0 Cited by
Patent Information
- Application Number
- TW114141893
- Authority / Receiving Office
- TW · TW
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-10-30
- Filing Date
- 2025-10-29
- Publication Date
- 2026-09-01
Smart Images
Figure TWG2TA001074531_001 
Figure TWG2TA001074531_002 
Figure TWG2TA001074531_003
Abstract
The disclosure relates to adeno-associated (AAV) particles comprising viral genomes encoding syntaxin-binding protein-1 (STXBP1), compositions comprising said AAV particles, and methods for making or delivering said AAV particles to a cell or subject. The AAV particles, compositions, and methods of the present disclosure are useful for the treatment of subjects who have, have been diagnosed with having, or are at risk of having a STXBP1-related disorder, e.g., STXBP1 Developmental and Epileptic Encephalopathy (DEE) and / or other STXBP1-related disorders, or at least one symptom thereof.
Need to check novelty before this filing date? Find Prior Art