Camp4 Therapeutics (CAMP) Corporate presentation summary

Strategic focus and platform technology

• Pioneering RNA medicines to increase targeted gene expression using proprietary RAP PlatformⓇ, which catalogs thousands of regulatory RNAs (regRNAs) and generates ASO candidates for upregulation of gene expression.

• RAP PlatformⓇ leverages machine learning and large-scale genomic datasets to identify and drug regRNAs across diverse cell and tissue types.

• Platform validated through strategic partnerships, including a collaboration with GSK for neurodegenerative and kidney disease targets.

Lead program and clinical pipeline

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

• CMP-002 is designed to increase SYNGAP protein levels, restore function, and improve symptoms; preclinical models show reversal of disease phenotype and significant protein upregulation.

• CMP-002 demonstrated efficacy in humanized mice and non-human primates, with broad brain distribution and well-tolerated intrathecal administration.

• Anticipates advancing CMP-002 to a global Phase 1/2 study in patients as early as the second half of 2026.

• Pipeline includes additional undisclosed CNS and metabolic disease programs, with ongoing discovery and development.

Unmet need and market opportunity

• SYNGAP1 disorder causes intellectual disability, epilepsy, severe behavioral and sleep problems, and high caregiver burden; current treatments are non-specific and symptom-focused.

• Prevalence estimated at ~21,000 patients across US and EU5, with diagnosis rates expected to rise due to expanded genetic testing and awareness.

• Multi-billion dollar commercial potential supported by prevalence, high unmet need, and lack of disease-modifying therapies.