Progeria Research Foundation Partners with Forge Biologics to Advance CRISPR Gene Therapy for Rare Aging Disease
核心洞察
The Progeria Research Foundation (搜索) has partnered with Forge Biologics (搜索) to manufacture SamPro-2 (搜索), an investigational CRISPR-based gene therapy designed to correct the genetic mutation causing Hutchinson-Gilford Progeria Syndrome (搜索).
SamPro-2 (搜索) uses base editing technology packaged in an AAV9 capsid to target the single DNA letter change in the lamin A gene (搜索) that leads to production of the toxic protein progerin (搜索).
The manufacturing agreement supports IND-enabling studies and represents a critical step toward clinical trials for this potentially curative treatment for children with the ultrarare fatal disease.
The Progeria Research Foundation (搜索) has entered into a manufacturing partnership with Forge Biologics (搜索) to advance SamPro-2 (搜索), an investigational CRISPR-based gene therapy designed to treat Hutchinson-Gilford Progeria Syndrome (搜索), a rare and fatal genetic disease that causes rapid aging in children.
The manufacturing agreement announced in March 2026 brings together PRF's decades-long research program with Forge's integrated gene therapy capabilities, including process development, cGMP manufacturing, and regulatory consultation. Forge will provide manufacturing services for Investigative New Drug (IND)-enabling studies with SamPro-2 (搜索), marking a critical step toward clinical development.
Revolutionary Gene Editing Approach
SamPro-2 (搜索) represents a novel therapeutic strategy that uses adeno-associated virus (AAV) as a delivery vehicle to deliver a base editing approach designed to correct the single DNA base mutation in the lamin A gene (搜索) that causes Progeria (搜索). The therapy is designed to permanently correct the Progeria genetic mutation at its source 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 (搜索).
"The era of Progeria (搜索) gene therapy has arrived. Our hope is that SamPro-2 (搜索) 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 (搜索), whose manufacturing expertise is essential to move this work from the laboratory towards clinical trials."
Distinguished Research Team
The development effort is led by a distinguished team of researchers collectively known as the Progeria (搜索) Gene Team. The team includes 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 (搜索), 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 (搜索); and the late Sammy Basso, M.S., former scientist, advocate, and enduring inspiration whose legacy continues to guide this work.
Manufacturing Partnership Details
Forge Biologics (搜索), a member of Ajinomoto Bio-Pharma Services (搜索), operates a 200,000 square foot facility called the Hearth in Columbus, Ohio, which houses 20 custom-designed cGMP suites with 20,000L of bioreactor capacity. The company'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.
"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 (搜索). "The Progeria Research Foundation (搜索) 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."
Path to Potential Cure
SamPro-2 (搜索) is being advanced as part of PRF's Path to Cure Progeria (搜索) initiative, which aims to determine whether a one-time gene-editing therapy can offer a durable, potentially curative treatment. The Progeria Research Foundation (搜索) has been the driving force behind every major scientific breakthrough in the field, from discovery of the gene that causes the disease to the first FDA-approved treatment, lonafarnib, and now to the advancement of gene-editing approaches.
The foundation was founded by the family of Sam Berns after his diagnosis in 1999 and has enabled or led every major scientific breakthrough in Progeria (搜索) research through rigorous science, global research infrastructure, and close partnership with the worldwide patient community.