CAMP4 Therapeutics Corporation is a clinical-stage biopharmaceutical company. The Company is focused on the discovery and development of regulatory RNA-based therapeutics with the goal of upregulating gene expression and restoring healthy protein levels to treat a range of genetic diseases. It is leveraging its RAP Platform to advance a pipeline of programs focused on metabolic and central nervous system (CNS) disorders with validated disease biology and potential market opportunities due to the significant unmet need of affected patients. Its lead product candidate, CMP-CPS-001, has the potential to be the first disease-modifying therapy for the treatment of urea cycle disorders. CMP-CPS-001 is designed to improve urea cycle activity by amplifying expression of CPS1, a key enzyme that catalyzes the first step of the urea cycle, by binding to a CPS1-specific regRNA. Its CNS development program, CMP-SYNGAP, is focused on addressing the underlying cause of SYNGAP1-related disorders.