We had the pleasure of supporting the International Porphyrias Symposium 2025 as a Sponsor. The event, held in Washington, DC from October 9 to 12 and organized by the United Porphyrias Association, brought together leading U.S. and international experts to discuss the clinical manifestations of each porphyria and the latest advances in diagnosis, management, and treatment.
On Saturday, October 11, our Chief Scientific Officer Oscar Millet presented an update on Congenital Erythropoietic Porphyria (CEP), sharing results from the Phase 1 clinical trial in which ATL-001 was administered for the first time to 42 healthy volunteers, demonstrating its safety and tolerability. He also provided insights on the upcoming Phase 1/2 clinical trial, which will be the first to administer ATL-001 to adult patients living with CEP.
The session also featured patient testimonials and a discussion on the psychosocial impact of CEP, offering a comprehensive view of the disease from both scientific and human perspectives.
During the event, Oscar Millet had the opportunity to meet again with Fide Mirón, a CEP patient advocate, as well as with Robert J. Desnick, PhD, MD, D.Sc. (Hon), Angelika Erwin and Hetanshi Naik, who are actively involved in the design and execution of the upcoming Phase 1/2 clinical trial.
It was a fantastic opportunity to share progress, engage with the porphyria community, and continue advancing toward a new treatment for this ultra-rare disease. Thank you to everyone who made this event possible!