Camp4 Therapeutics Corp

Camp4 Therapeutics Corp is a clinical-stage biopharmaceutical company focused on discovering and developing a new class of RNA-targeting therapeutics. The company aims to upregulate gene expression and restore healthy protein levels to treat a wide range of genetic diseases. Its lead product candidate, CMP-002, is being developed as a potential disease-modifying therapy for SYNGAP1-related disorder, also known as SYNGAP1. This disorder is a severe developmental and epileptic encephalopathy marked by seizures, developmental delays, and cognitive impairments. SYNGAP1 is caused by haploinsufficiency of the SYNGAP1 gene, where a mutation in one gene copy reduces functional SYNGAP protein levels by up to 50%. The company estimates approximately 21,000 individuals live with SYNGAP1 in the United States and the five largest European markets, with no approved disease-modifying therapies currently available. CMP-002 is a novel, intrathecally delivered antisense oligonucleotide (ASO) designed to increase SYNGAP1 gene expression at the transcriptional level, potentially increasing SYNGAP protein levels to therapeutic amounts. Preclinical studies showed that intracerebroventricular injection of CMP-002 restored SYNGAP protein levels to near normal range in haploinsufficient mice carrying a single copy of the human SYNGAP1 gene after one dose, and rescued motor and spatial learning defects after two doses. Biweekly intrathecal injections of CMP-002 in cynomolgus monkeys were well tolerated and significantly increased SYNGAP protein levels across multiple disease-relevant brain regions, with dose-linear increases of CMP-002 in these regions. The company has initiated Good Laboratory Practice (GLP) toxicology studies for CMP-002 to support clinical trial applications. Product development is supported by its proprietary RAP Platform, which identifies and characterizes regulatory RNAs (regRNAs) involved in gene activation and suppression.