CMP-SYNGAP-01

CMP-SYNGAP-01 is an investigational antisense oligonucleotide (ASO) therapy developed by CAMP4 Therapeutics for the treatment of SYNGAP1-related neurodevelopmental disorders. These disorders are caused by haploinsufficiency or loss-of-function mutations in the SYNGAP1 gene, leading to insufficient levels of synaptic Ras GTPase activating protein 1 (SynGAP), which is critical for normal brain function. CMP-SYNGAP-01 targets a regulatory RNA sequence mapped to a SYNGAP1 gene regulatory region, with the goal of upregulating expression from the remaining functional allele and restoring SynGAP protein levels. Preclinical studies in haploinsufficient mice demonstrated that intracerebroventricular administration of CMP-SYNGAP-01 restored SynGAP protein to near-normal levels and rescued motor and cognitive deficits. In non-human primates, biweekly intrathecal injections led to approximately a 1.5-fold increase in SynGAP protein across multiple disease-relevant brain regions. The drug has shown favorable tolerability in preclinical models and represents a novel approach for addressing genetic diseases characterized by insufficient protein production[3][5][6][8].

Other names

SYNGAP (Camp4 Therapeutics)