WATERTOWN, Mass. — September 8, 2026
CAMP4 Therapeutics Corporation announced plans to host a virtual Analyst Day on September 28, 2026, focused on CMP-002 and its development strategy for SYNGAP1-related disorder. The event will feature presentations and a question-and-answer session led by CAMP4’s executive leadership, with participation from patient advocates and pediatric neurology expert Stéphane Auvin, M.D., Ph.D. The company plans to discuss the significant unmet clinical need associated with SYNGAP1-related disorder, provide details on the design of its planned first-in-human Phase 1/2 clinical trial of CMP-002, and provide an update on its early discovery pipeline. CAMP4 expects the CMP-002 clinical program to initiate in Q4 2026, making the upcoming Analyst Day an important corporate and development milestone as the company moves its lead regulatory RNA-targeting therapeutic toward clinical evaluation.
CMP-002 Targets SYNGAP1 Protein Restoration
CMP-002 is designed as a potentially first-in-class disease-modifying therapy for SYNGAP1-related disorder, a rare central nervous system condition caused by mutations in the SYNGAP1 gene that result in approximately 50% of normal SYNGAP protein levels. CAMP4 is developing CMP-002 as an antisense oligonucleotide (ASO) designed to bind a SYNGAP1-specific regulatory RNA and increase expression of the SYNGAP1 gene. The candidate is intended to restore SYNGAP protein toward near-normal levels rather than simply managing individual symptoms. Administered intrathecally, CMP-002 has generated preclinical evidence of dose-dependent increases in SYNGAP protein expression in patient-derived neurons, as well as improvements in disease-relevant behavioral and seizure measures in animal models. Non-human primate studies have also demonstrated broad brain distribution and significant SYNGAP protein upregulation, providing the preclinical foundation for advancing the program toward human testing.
Phase 1/2 Program Targets Major Unmet Need
CAMP4’s planned Phase 1/2 trial comes as SYNGAP1-related disorder remains without an approved disease-modifying therapy, despite substantial neurological and developmental burden. The disorder is associated with intellectual disability, epilepsy, behavioral problems, sleep disturbances and significant limitations in communication. CAMP4 estimates that more than 10,000 individuals in the United States are affected. The company’s Analyst Day is expected to provide additional visibility into the clinical trial design and development strategy before first-in-human testing begins. Participation from a leading pediatric neurologist and patient advocates is intended to provide perspectives on both the clinical landscape and the experience of families affected by the disorder. For CAMP4, establishing a clinical development pathway for CMP-002 represents an important step in translating its regulatory RNA technology into a disease-modifying treatment approach for a genetically defined CNS disorder.
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CAMP4 Advances Regulatory RNA Therapeutics Platform
CMP-002 also serves as a clinical demonstration of CAMP4’s broader RAP Platform® strategy, which is designed to increase healthy protein production by targeting regulatory RNAs involved in gene-expression control. Rather than directly replacing defective genetic material, CAMP4’s ASO approach is intended to amplify endogenous gene expression by targeting regRNAs that regulate transcriptional activity. The company is applying this strategy to haploinsufficient and recessive partial loss-of-function disorders, where increasing protein levels could potentially provide meaningful therapeutic benefit. CAMP4 says its platform has identified more than 1,200 genetic disorders in which modest increases in protein expression may have clinical potential. As CMP-002 moves toward Phase 1/2 development, the company expects to use clinical and translational insights from the program to advance its broader discovery pipeline. The September Analyst Day therefore represents both a CMP-002 development update and an opportunity for CAMP4 to outline how its regulatory RNA platform could support a wider portfolio of genetic disease programs.
Source: CAMP4 Therapeutics, ,press release
