Camp4 Therapeutics delivers three presentantions on SYNGAP1-related disorders

CAMP4 Therapeutics (CAMP) delivered three oral presentations on its SYNGAP1-related disorders and Urea Cycle Disorders, or UCDs, programs and shared favorable safety and pharmacokinetics data from the ongoing Phase 1 trial of CMP-CPS-001 in healthy volunteers at the 28th Annual Meeting of the American Society of Gene and Cell Therapy, taking place in New Orleans, May 13-17. Key findings for each program are as follows: In haploinsufficient mice carrying a single copy of the human SYNGAP1 gene, intracerebroventricular, or ICV, injection of CMP-SYNGAP-01, a development candidate targeting a regulatory RNA sequence mapped to a SYNGAP1 gene regulatory region, resulted in: Restored SYNGAP1 protein levels to near normal range after a single dose; rescue of motor defects and spatial learning defects following two doses, in NHPs, biweekly intrathecal injections of CMP-SYNGAP-01 resulted in a ~1.5-fold increase in SYNGAP1 protein levels across multiple brain regions clinically relevant to the disease; Dose-linear increase in CMP-SYNGAP-01 in disease-relevant brain regions CMP-SYNGAP-01 was well tolerated. The presentations can be accessed on the CAMP4 website.