Camp4 Therapeutics at Cantor conference: pushing first SYNGAP1 trial

On September 9, 2026, Camp4 Therapeutics (CAMP) highlighted its lead SYNGAP1 program and a regRNA platform at the 12th Annual Cantor Fitzgerald Global Healthcare Conference. The company outlined meaningful regulatory progress and a clear clinical path for the rare genetic disease. SYNGAP1 is caused by haploinsufficiency and presents with seizures, learning problems, and developmental delays.

Camp4 is the first company developing a therapy for SYNGAP1, and it has approvals for its Phase I/IIa multiple ascending dose study in Australia, Argentina, and the United Kingdom, with FDA review ongoing. The trial, set for Q4 2024, will enroll about 30 participants across three dose cohorts using a placebo-controlled design with crossover options.

Camp4's regRNA platform aims to raise gene expression rather than silence it, potentially applicable to thousands of targets across haploinsufficient diseases. A collaboration with GSK on two non-DEE targets offers milestone and royalty potential while Camp4 focuses on SYNGAP1. CEO Josh Mandel-Brehm emphasized the genetic nature of the disease and the need for an approved treatment, stating that awareness of SYNGAP1 has risen sharply over the past year.

The company expects to start the trial with older patients and then progress to younger children, capturing a broad set of outcomes in addition to seizure counts.