CAMP4 Therapeutics Corporation announced on April 28, 2025, that it will deliver three oral presentations at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in May 2025. These presentations will highlight new preclinical data from its Urea Cycle Disorders (UCDs) and SYNGAP1-related disorders programs.
The presentations will showcase the potential of regulatory RNA-targeting to increase protein levels by upregulating gene expression. This includes data on CMP-CPS-001 for UCDs and CMP-SYNGAP-01 for SYNGAP1-related disorders.
Additionally, interim safety data, including patient demographic data from all Single Ascending Dose (SAD) cohorts of the first-in-human Phase 1 clinical trial of CMP-CPS-001 in healthy volunteers, will be presented. This provides an important update on the safety profile of the lead clinical candidate.
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