Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
STXBP1 ASO
- Development stage
- Preclinical
- Lead developer
- Quiver Bioscience
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal, Stereotactic Intracranial Injection
01
Overview
STXBP1 ASO is an antisense oligonucleotide therapeutic candidate being developed by Quiver Bioscience for the treatment of STXBP1-related developmental and epileptic encephalopathy 4 (DEE4). The drug targets STXBP1 mRNA, which encodes the syntaxin-binding protein 1 (also known as Munc18-1), a protein critical for synaptic vesicle fusion and neurotransmitter release. Mutations in STXBP1 typically result in haploinsufficiency, leading to impaired neurotransmitter release and neuronal dysfunction. This ASO is designed to modulate STXBP1 mRNA levels or splicing to restore functional protein levels. The program is currently in the discovery phase and leverages Quiver's proprietary Genomic Positioning System (GPS) platform, which integrates human single-cell neuronal electrophysiology with AI/ML analytics for design and optimization.
- Other names
- STXBP1 antisense oligonucleotideSTXBP-1 antisense oligonucleotideSTXBP 1 antisense oligonucleotide
02
Targets
STXBP1 (Syntaxin-binding protein 1)
Beyond the preview
Go deeper on STXBP1 ASO.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on STXBP1 ASO.