The Progeria Research Foundation and Forge Biologics Announce Manufacturing Partnership to Advance Gene Therapy for Children with Progeria

• Partnership marks a critical step toward clinical development of an AAV gene therapy using a base editing approach designed to target the genetic cause of Hutchinson-Gilford Progeria Syndrome

• Forge Biologics will provide process development and manufacturing expertise to support gene therapy advancement led by world-renowned researchers Leslie Gordon, David Liu, and Francis Collins

PEABODY, Mass. & COLUMBUS, Ohio--(BUSINESS WIRE)--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.”

About SamPro-2

SamPro-2 is an investigational in vivo gene-editing therapy being advanced by The Progeria Research Foundation (PRF) as part of its Path to Cure Progeria initiative. SamPro-2 is designed to correct the underlying genetic cause of Hutchinson-Gilford Progeria Syndrome (Progeria) using precision base-editing technology, packaged in an AAV9 capsid, to correct the single DNA letter change that leads to production of the toxic protein progerin.

About The Progeria Research Foundation

The Progeria Research Foundation (PRF) is the driving force behind the global effort to understand, treat and ultimately cure Hutchinson-Gilford Progeria Syndrome (Progeria), a rare and fatal genetic disease that causes rapid aging in children. Founded by the family of Sam Berns after his diagnosis in 1999, PRF has enabled or led every major scientific breakthrough in the field, from discovery of the gene that causes the disease to the first FDA-approved treatment, lonafarnib, to the advancement of gene-editing approaches now in development. Through rigorous science, global research infrastructure and close partnership with the worldwide patient community, PRF is advancing next-generation therapies, expanding diagnosis and care through its Find the Children initiative, and leading the Path to Cure Progeria program to determine whether a one-time gene-editing therapy can offer a durable, potentially curative treatment. For more information and to support PRF’s mission, please visit www.progeriaresearch.org.

About Forge Biologics

Forge Biologics, a member of Ajinomoto Bio-Pharma Services, is a gene therapy contract development and manufacturing organization (CDMO) enabling access to life-changing gene therapies by bringing them from concept to reality. Forge’s 200,000 square foot facility, the Hearth, is headquartered in Columbus, Ohio, and houses 20 custom-designed cGMP suites with 20,000L of bioreactor capacity. Forge’s end-to-end, scalable plasmid and AAV manufacturing services include research-grade manufacturing, process and analytical development, cGMP manufacturing, fill and finish, and integrated regulatory support to help accelerate the timelines of transformative medicines for patients with genetic diseases. To learn more, visit www.forgebiologics.com.