Camp4 Therapeutics Corp

CAMP4 Therapeutics Corporation (Nasdaq: CAMP4) is a clinical-stage biopharmaceutical company pioneering the discovery and development of RNA-targeting therapeutics designed to upregulate gene expression and restore healthy protein levels for treating genetic diseases. The company operates through its proprietary RAP Platform, which identifies and characterizes regulatory RNAs that control protein-coding genes, enabling the design of antisense oligonucleotide candidates to amplify messenger RNA expression in a specific and controllable manner.

The company's lead product candidate, CMP-002, targets SYNGAP1-related disorder, a severe developmental and epileptic encephalopathy caused by haploinsufficiency of the SYNGAP1 gene. CMP-002 is designed as an intrathecally delivered antisense oligonucleotide intended to increase SYNGAP1 gene expression and restore protein levels. Preclinical studies demonstrated dose-dependent increases in SYNGAP protein levels in humanized mouse models and broad brain distribution with improved behavioral phenotypes in primates. The company has initiated Good Laboratory Practice toxicology studies and plans to initiate a global Phase 1/2 clinical trial as early as the second half of 2026.

Beyond its lead program, CAMP4 is advancing CMP-001 for urea cycle disorders, which completed a Phase 1 clinical trial in healthy volunteers. The company has also established a strategic research collaboration and license agreement with GlaxoSmithKline to extend its RAP Platform application beyond the central nervous system into neurodegenerative and kidney disease indications, with potential milestone payments up to $440 million.

The company's therapeutic focus centers on rare and prevalent haploinsufficient diseases where modest increases in protein expression could provide clinical benefit. CAMP4 estimates approximately 21,000 individuals living with SYNGAP1 in the United States and five largest European markets, with no currently approved disease-modifying therapies available. The company does not operate manufacturing facilities and relies on third-party contract manufacturers for production. Founded on pioneering transcription regulation research, CAMP4 maintains proprietary intellectual property including licensed patents from the Whitehead Institute for Biomedical Research. More information is available at www.camp4therapeutics.com.