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Disease Areas

At CAMP4, we believe every patient deserves hope.

Our mission is to develop medicines that increase critical protein levels in the body by targeting regulatory RNAs (regRNAs), offering potential new treatment options for patients with serious disorders who need more effective therapies.

Many serious diseases are caused by insufficient production of essential proteins. Even a modest increase in protein levels has the potential to make a meaningful difference in patient health. By precisely modulating regRNAs, CAMP4’s therapies are designed to restore levels of protein expression and address the root cause of genetic diseases. This approach may offer new hope for patients and families who have long been waiting for effective disease-modifying therapies.

SYNGAP1-Related Disorder

Resources

CURE SYNGAP1 – Dedicated to improving the quality of life of SRD patients through the research and development of treatments, therapies and support systems.

    What is SYNGAP1-Related Disorder?

    SYNGAP1-related disorder (SRD) is a rare genetic disease caused by harmful mutations in the SYNGAP1 gene. These mutations frequently result in inadequate levels of SYNGAP protein, which is essential for healthy brain development and function. Low SYNGAP protein levels disrupt normal brain signaling, leading to a wide range of developmental and neurological challenges.

    Our Approach

    CMP-002 is designed to upregulate SYNGAP1 expression by targeting a regulatory RNA sequence mapped by CAMP4’s RAP Platform®. In preclinical studies, CMP-002 restores SYNGAP protein levels and significantly improved behavioral outcomes in animal models.

    Clinical Trials

    Expect to initiate as early as the second half of 2026.