CAMP4 Therapeutics has secured approval from Australia’s regulator to begin a first-in-human trial of its SYNGAP1-targeting antisense oligonucleotide. The study will enroll at least 30 patients aged 2-18 across multiple sites, use a double-blind, placebo-controlled design with crossover, and marks the first human use of ASOs for SYNGAP1. Argentina approved a trial; U.S. regulatory discussions are planned, with more details at Sept 28 investor day.
CAMP4 priced its IPO at $11.00, raising $75 million. CMP-CPS-001 has FDA Orphan Drug designations for urea cycle disorders. CAMP4 partners with BioMarin to target regulatory RNA for new therapies. Analysts note CAMP4 trades at low market cap despite innovative platform. Piper Sandler forecasts $1 billion in revenues for CMP-CPS-001 by 2036.
