CAMP4 reports SYNGAP1 protein increase in NHPs and favorable Phase 1 safety data for UCD program at ASGCT

Camp4 Therapeutics Corp

CMP-SYNGAP-01 restored SYNGAP1 protein to near-normal in haploinsufficient mice and rescued motor/learning defects.
In NHPs, biweekly intrathecal CMP-SYNGAP-01 achieved ~1.5-fold SYNGAP1 increase in relevant brain regions.
Phase 1 SAD data for CMP-CPS-001 in 48 healthy volunteers: well tolerated, no serious AEs, dose-dependent PK.
UCD preclinical: CMP-CPS-001 increased ureagenesis up to 40% in NHPs and reduced ammonia in mice.
Company plans to expand Phase 1 into OTC heterozygotes with reduced urea cycle function.

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