CAMP4 Therapeutics Corporation delivered three oral presentations at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) on May 16, 2025. The presentations highlighted compelling translational data from its SYNGAP1-related disorders program and favorable safety and pharmacokinetics data from the ongoing Phase 1 trial of CMP-CPS-001 in healthy volunteers.
For the SYNGAP1-related disorders program, CMP-SYNGAP-01 demonstrated an increase in SYNGAP1 protein levels in haploinsufficient mice, which rescued multiple SYNGAP1-dependent behavioral phenotypes. Importantly, CMP-SYNGAP-01 administration also led to a significant increase in SYNGAP1 protein levels in relevant brain regions in non-human primates following intrathecal administration.
Regarding the Urea Cycle Disorders (UCDs) program, patient safety and pharmacokinetic data from the single ascending dose (SAD) cohorts of the first-in-human Phase 1 clinical trial of CMP-CPS-001 in healthy volunteers were reviewed. The data highlighted the drug's favorable safety profile, supporting its continued development.
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