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Regulatory approvals and multi-country trial progress are near-term catalysts; potential data readouts and U.S. discussions could lift sentiment and valuation for CAMP4's SYNGAP1 program.
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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.
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