Business
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 Urea Cycle Disorders Program at the 28th American Society of Gene and Cell Therapy Annual Meeting
Haploinsufficient SYNGAP1 mice treated with CMP-SYNGAP-01 demonstrated an increase in SYNGAP1 pro...
About this update from Canadian Goldcamps Corp
[{"type":"text","content":"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 Urea Cycle Disorders Program at the 28th American Society of Gene and Cell Therapy Annual Meeting\n\n\n\n\n Haploinsufficient SYNGAP1 mice treated with CMP-SYNGAP-01 demonstrated an increase in SYNGAP1 protein levels; treatment rescued multiple SYNGAP1-dependent behavioral phenotypes\n \n\n\n\n CMP-SYNGAP-01 administration led to a significant increase in SYNGAP1 protein levels in relevant brain regions in non-human primates (NHPs)\n \n\n\n\n Patient safety and pharmacokinetic data from single ascending dose (SAD) cohorts of the first-in-human Phase 1 clinical trial of CMP-CPS-001 in healthy volunteers highlighted\n \n\n\n CAMBRIDGE, Mass., May 16, 2025 (GLOBE NEWSWIRE) -- CAMP4 Therapeutics Corporation (“CAMP4”) (Nasdaq: CAMP), a clinical-stage biopharmaceutical company developing a pipeline of regulatory RNA-targeting therapeutics designed to upregulate gene expression with the goal of restoring healthy protein levels to treat a broad range of genetic diseases, today delivered three oral presentations on its SYNGAP1-related disorders and Urea Cycle Disorders (UCDs) programs and shared favorable safety and pharmacokinetics data from the ongoing Phase 1 trial of CMP-CPS-001 in healthy volunteers at the 28\n \n th\n \n Annual Meeting of the American Society of Gene and Cell Therapy, taking place in New Orleans, May 13 – 17, 2025.\n \n\n “Patients living with SYNGAP1-related disorders and UCDs currently face a critical dearth of disease-modifying treatment options to manage their condition,” said Dan Tardiff, Ph.D., Senior Vice President, Head of Discovery at CAMP4. “These proof-of-mechanism data indicate CMP-SYNGAP-01 can restore SYNGAP1 protein levels to mitigate disease-relevant phenotypes in haploinsufficient mice and increase SYNGAP1 protein in disease-relevant brain regions in non-human primates when delivered by the clinical route of administration. Additionally, our UCD clinical candidate is well tolerated, and we are preparing to evaluate CMP-CPS-001 in OTC heterozygotes, a population with reduced urea cycle function. We’re excited to continue pioneering our novel approach of u...