CAMP4 Therapeutics is a clinical-stage biopharmaceutical company developing RNA-targeting therapeutics designed to increase gene expression — rather than silence or edit genes — in patients with genetic diseases caused by insufficient protein production. Its lead program, CMP-002, is an antisense oligonucleotide (ASO) delivered intrathecally that targets SYNGAP1-related disorder, a rare and severe neurological disease characterized by seizures, intellectual disability, and developmental delays, affecting an estimated 21,000 patients in the U.S. and major European markets. CAMP4 is conducting toxicology studies and targets a Phase 1/2 trial initiation as early as the second half of 2026. A second program, CMP-001, targeting urea cycle disorders, completed a Phase 1 trial but showed no conclusive efficacy signal; CAMP4 paused investment in CMP-001 in Q2 2025 and is seeking a partner to advance it. CAMP4's core technology is its RAP Platform, which maps regulatory RNAs that control gene expression and uses proprietary machine learning to design ASOs that amplify transcription of target genes. The company has two potential revenue paths: developing and commercializing drugs independently, and licensing the RAP Platform to larger pharma partners. In December 2025, CAMP4 signed a collaboration agreement with GSK covering neurodegenerative and kidney disease, receiving a $17.5M upfront payment and eligibility for up to $440M in milestones plus royalties. CAMP4 has no approved products, outsources all manufacturing, and currently depends on partnership economics for near-term cash flows.