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Psd3-ASO
- Development stage
- Preclinical
- 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
Psd3-ASO is an antisense oligonucleotide (ASO) designed to reduce the expression of the *Psd3* (Pleckstrin and Sec7 domain containing 3) gene. *Psd3* was identified as a candidate modifier gene for Dravet Syndrome, a developmental and epileptic encephalopathy often associated with mutations in the *SCN1A* gene. In preclinical studies using the *Scn1a*+/- mouse model, Psd3-ASO is administered via intracerebroventricular (ICV) injection to investigate its potential to improve survival and modify seizure phenotypes. Research presented at the AES Annual Meeting 2025 indicated that while Psd3-ASO treatment showed a trending improvement in median survival in certain mouse strains, it did not significantly alter seizure thresholds in hyperthermia-induced seizure assays.
- Other names
- ASO targeting Psd3
02
Targets
GEF/GAP (Ras GEF / GAP family)
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