Yuri Maricich, MD, Chief Medical Officer of CAMP4 Therapeutics, discusses the company’s current drug development programs.
CAMP4 is a biopharmaceutical company focused on utilizing RNA to develop novel treatments that increase gene expression in patients with rare genetic conditions.
CAMP4 is currently working on CMP-CPS-001, a novel investigational therapy designed to address the underlying cause of urea cycle disorders (UCDs) by regulating genes associated with the condition. The first clinical trial of this program was initiated in March 2024 with a phase 1 single-ascending dose study in healthy volunteers. Following the completion of this trial, a multiple-ascending dose study was initiated.
Recently, the company announced a phase 1b expansion of this study in an ornithine transcarbamylase (OTC) heterozygote population. Upon completion of this extension, the goal is to transition into a phase 3 registrational clinical trial in the second half of 2026.
A second treatment is being developed for SYNGAP1-related disorders. While CMP-SYNGAP is not yet in clinical trials, encouraging results have come from testing in animal models. The company has plans to present data at this year’s American Society of Gene & Cell Therapy meeting (ASGCT 2025).
For more information on CAMP4 Therapeutics and their work, click here.
To learn more about rare metabolic conditions, visit https://checkrare.com/diseases/metabolic-disorders/
