Camp4 Therapeutics (CAMP) Corporate presentation summary

Scientific and technological platform

• Pioneering RNA medicines to increase targeted gene expression using proprietary RAP PlatformⓇ, which catalogs thousands of regulatory RNA (regRNA) targets and generates ASO candidates to upregulate gene expression.

• RAP PlatformⓇ leverages genome-wide chromatin and RNA analyses, machine learning, and large-scale sequencing to identify and optimize regRNA targets for drug development.

• ASOs disrupt interactions between transcription factors and regRNAs, enabling increased gene expression of targeted genes.

Lead program and disease focus

• Lead program CMP-002 targets SYNGAP1-related disorder, a haploinsufficient CNS disease with over 10,000 US patients and no approved disease-modifying therapies.

• CMP-002 is designed to increase SYNGAP protein levels, restore function, and improve symptoms, with preclinical data showing reversal of disease phenotypes in humanized mice and significant protein upregulation in primates.

• GLP toxicology studies are ongoing, with a global Phase 1/2 clinical trial anticipated to start as early as the second half of 2026.

Unmet need and patient impact

• SYNGAP1 disorder causes intellectual disability in all patients, seizures in ~85%, severe behavioral problems, and high lifelong care burden.

• No disease-modifying therapies exist; current treatments are symptomatic and often require polypharmacy.

• Diagnostic journey has improved from ~5 years to ~1 year due to increased awareness and genetic testing.