CAMP4 Presents Translational Data from SYNGAP1-Related Disorders Program Showcasing Increased Protein in Non-Human Primates and Reviews Preclinical and Detailed Single Ascending Dose Safety Data from

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May 16, 2025
  • CAMP4 Therapeutics presents promising translational data for SYNGAP1 and Urea Cycle Disorders programs at the ASGCT Annual Meeting.
  • SIGNIFICANT protein increase observed in SYNGAP1 program in both mouse models and non-human primates.
  • Phase 1 trial for UCDs shows CMP-CPS-001 is well-tolerated with no serious adverse events.

CAMP4 Therapeutics (CAMP, Financial) has unveiled compelling translational data from its SYNGAP1-related disorders and Urea Cycle Disorders (UCDs) programs at the 28th Annual Meeting of the American Society of Gene and Cell Therapy. Utilizing their regulatory RNA-targeting platform, CAMP4 aims to tackle genetic disorders marked by insufficient protein production.

In the SYNGAP1-related disorders program, CMP-SYNGAP-01 demonstrated substantial efficacy in both haploinsufficient mice and non-human primates (NHPs). In treated mice, SYNGAP1 protein levels were restored, rescuing key behavioral deficits. Notably, NHPs receiving intrathecal injections exhibited an approximate 150% SYNGAP1 protein increase across several critical brain regions, with no safety concerns reported.

For the UCDs program, preclinical data highlighted the capacity of CMP-CPS-001 to generate dose-dependent ammonia reductions lasting around four weeks. In NHPs, ureagenesis increased by up to 40%. Phase 1 clinical trial results from single ascending dose cohorts revealed CMP-CPS-001 was well tolerated, with no serious adverse events, and exhibited a dose-dependent increase in exposure.

CAMP4 is progressing rapidly, having completed dosing in multiple dose cohorts, with plans to extend studies into OTC heterozygotes. This initiative represents a significant stride in addressing two serious genetic conditions with high unmet medical needs.

By focusing on regulatory RNA to enhance gene expression, CAMP4's approach offers a differentiated therapeutic strategy compared to traditional gene replacement methods. Their advancements indicate substantial potential for this platform to address genetic disorders characterized by haploinsufficiency or recessive loss of function.

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I/We may personally own shares in some of the companies mentioned above. However, those positions are not material to either the company or to my/our portfolios.