Advancing a Gene-Upregulating Oligo for SYNGAP1: From Preclinical Validation to Clinical Entry Preparation
- SYNGAP is a synaptic protein that when mutated results in haploinsufficiency characterized by developmental delays, behavioral issues, limited communication, and epilepsy
- CMP-002 is an antisense oligonucleotide that targets a noncoding regulatory RNA to increase SYNGAP1 transcription, which ameliorates multiple phenotypes in a mouse model and increases SYNGAP protein in monkeys when administered by intrathecal injection
- Preparation is underway to enable the assessment of CMP-002 in a global Phase 1/2 clinical study in SYNGAP1-related disorder patient