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Stoke STXBP1 ASO
- Development stage
- Preclinical
- Lead developer
- Stoke Therapeutics
- 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
01
Overview
Stoke Therapeutics is developing an antisense oligonucleotide (ASO) for the treatment of STXBP1-related developmental and epileptic encephalopathy (STXBP1-DEE) and other CNS diseases caused by STXBP1 haploinsufficiency. The program utilizes Stoke's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) technology, which is designed to restore functional protein levels by modulating the splicing of pre-mRNA. Specifically, the ASO targets and prevents the inclusion of non-productive 'poison' exons, thereby increasing the production of functional Syntaxin-binding protein 1 from the healthy allele. This approach aims to address the underlying genetic cause of the disorder, which typically presents with severe intellectual disability, motor impairment, and refractory epilepsy.
- Other names
- STXBP1 TANGO ASOSTXBP-1 TANGO ASOSTXBP 1 TANGO ASO
02
Targets
STXBP1 (Syntaxin-binding protein 1)
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