- 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.
