The Progeria Research Foundation (“PRF”), a nonprofit research organization dedicated to developing treatments and the cure for Hutchinson-Gilford Progeria Syndrome (“Progeria”), and Forge Biologics, (“Forge”), a leading manufacturer of gene therapies and member of the Ajinomoto Bio-Pharma Services group, today announced a manufacturing agreement to support the development and manufacturing of SamPro-2, PRF’s investigational gene therapy for children and young adults living with Progeria, an ultrarare and fatal genetic disease characterized by rapid aging.

The manufacturing agreement brings together PRF’s decades-long, research-driven Progeria program with Forge’s integrated gene therapy capabilities including process development, cGMP manufacturing, FUEL™ platform technologies, and regulatory consultation. Forge will provide manufacturing services for Investigative New Drug (IND)-enabling studies with SamPro-2, a gene therapy that uses adeno-associated virus (AAV), a commonly utilized delivery vehicle in gene therapy, to deliver a base editing approach designed to correct the single DNA base mutation in the lamin A gene that causes Progeria.

“The era of Progeria gene therapy has arrived. Our hope is that SamPro-2 will give children and young adults with Progeria the longer, healthier lives they deserve,” said Leslie Gordon, M.D., Ph.D., co-founder and medical director of PRF and the mother of Sam Berns who had Progeria. “We are extremely grateful to be working with Forge Biologics, whose manufacturing expertise is essential to move this work from the laboratory towards clinical trials.”

PRF and its collaborators, collectively known as the Progeria Gene Team, have created SamPro-2, a CRISPR-based gene editing strategy designed to permanently correct the Progeria genetic mutation at its source. The effort is led by Dr. Gordon, a leading Progeria expert and clinical trialist; David R. Liu, Ph.D., Richard Merkin Professor and Director of the Merkin Institute for Transformative Technologies in Healthcare at the Broad Institute of MIT and Harvard, whose laboratory has been an international leader in the development of base editing technology; Francis S. Collins, M.D., Ph.D., Senior Research Advisor to the Progeria Research Foundation; and the late Sammy Basso, M.S., former scientist, advocate, and enduring inspiration whose legacy continues to guide this work.

“Behind every program like this are patients and families who have waited a long time for progress,” said John Maslowski, president and chief executive officer of Forge Biologics. “The Progeria Research Foundation and its Gene Team have shown extraordinary dedication to advancing this science, and we are honored to partner with them. At Forge, we bring that same level of care, expertise, and technical rigor to our manufacturing work as we help advance this program for patients.”