We are a clinical biopharmaceutical company pioneering the discovery and development of regulatory RNA-based therapeutics with the goal of upregulating gene expression and restoring healthy protein levels to a treat a broad range of genetic diseases. We are focused on metabolic and CNS (central nervous system) diseases. (Incorporated in Delaware)

We believe our lead product candidate, CMP-CPS-001, has the potential to be the first disease-modifying therapy for the treatment of the most prevalent urea cycle disorders, or UCDs. UCDs are a group of severe, inherited metabolic diseases caused by mutations in the genes that encode one or more of the eight enzymes and transporters necessary to convert ammonia into urea. The inability of the body to properly metabolize ammonia leads to the accumulation of toxic levels in circulation, ultimately resulting in severe health outcomes, such as neurologic disability, seizure and death. CMP-CPS-001 is designed to improve urea cycle activity by amplifying expression of carbamoyl phosphate synthetase 1, or CPS1, an enzyme that catalyzes the first step of the urea cycle, by binding to a CPS1-specific regRNA. Our preclinical studies have demonstrated that modulating the activity of the target regRNA increases expression of the CPS1 gene, resulting in increased CPS1 enzyme levels, which allows for more ammonia to be converted into urea, thereby lowering ammonia levels to normal, healthy ranges. These preclinical studies also demonstrated that CMP-CPS-001 can increase the level of, or upregulate, the production of multiple enzymes responsible for converting ammonia into urea, potentially allowing us to address more than 85% of patients with UCDs, which we refer to as our pan-UCD approach.

We are in the early stages of development and are evaluating CMP-CPS-001 in an ongoing Phase 1 clinical trial in healthy volunteers and expect to report data from all four cohorts of the single ascending dose, or SAD, portion of the trial in the first quarter of 2025 and from the multiple ascending dose, or MAD, portion of the trial in the second half of 2025. We are also leveraging our RAP Platform to advance our first preclinical program for the treatment of synaptic Ras GTPase activating protein 1, or SYNGAP1,-related disorders. We expect to initiate final Good Laboratory Practice, or GLP, toxicology studies in our SYNGAP1 program in 2025 to enable the filing of clinical trial applications.

(Note: Camp4 Therapeutics Corp. priced its IPO at $11.00 – $3.00 below the bottom of its $14.00-to-$16.00 price range – and increased the IPO’s size to 6.82 million shares – up from 5.0 million shares in the prospectus – to raise $75 million on Thursday night, Oct. 10, 2024. Background: Camp4 Therepeutics Corp. filed its S-1 on Sept. 20, 2024, without disclosing terms for its IPO. Estimated proceeds are $75.0 million. Background: Camp Therapeutics Corp. submitted confidential IPO documents to the SEC on June 14, 2024.)