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SYNGAP1 antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- CAMP4 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
SYNGAP1 antisense oligonucleotide is a preclinical-stage genetic medicine being developed by Quiver Bioscience, in collaboration with Quralis, for the treatment of developmental and epileptic encephalopathy (DEE). This antisense oligonucleotide (ASO) therapeutic is designed to modulate the expression of the SYNGAP1 gene, addressing the haploinsufficiency that characterizes SYNGAP1-related disorders. By leveraging Quiver's proprietary Genomic Positioning System (GPS) platform, which integrates human neuronal models with artificial intelligence and machine learning analytics, the program aims to restore functional levels of the Synaptic Ras GTPase-activating protein 1 (SYNGAP1) to alleviate neurodevelopmental symptoms, including epilepsy and intellectual disability.
- Other names
- SYNGAP antisense oligonucleotideSYNGAP1 ASOSYNGAP-1 ASOSYNGAP 1 ASOSYNGAP1 antisense oligonucleotideSYNGAP-1 antisense oligonucleotideSYNGAP 1 antisense oligonucleotide
02
Targets
SYNGAP1 (Synaptic Ras GTPase-activating protein 1)
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