Targeting Regulatory RNAs with Antisense Oligonucleotides for the Potential Treatment of SYNGAP1-Related Disorders
Time: 10:30 am
day: Day 2
Details:
- Antisense oligonucleotides target non-coding regulatory RNAs to boost gene expression in haploinsufficient CNS diseases
- Unique modality enabling functional protein restoration without gene editing or replacement
- Lead program in SYNGAP1-related disorders has disease-modifying potential to broadly address disease symptoms
- Preclinical data show efficacy in a humanized mouse model and increased SynGAP protein in non-human primates with GLP toxicology studies planned
