PRF Announces Manufacturing Partnership with Forge Biologics to Advance Progeria Gene Therapy!

PRF has entered into a manufacturing agreement with Forge Biologics to produce SamPro-2 – PRF’s investigational gene-editing therapy designed to correct the gene mutation that causes Progeria.

This is a defining step toward Progeria gene therapy clinical trials.

What is SamPro-2?
SamPro-2 is PRF’s gene editing therapy designed to correct the single DNA letter misspelling that causes Progeria.

DNA is the body’s instruction manual. In children and young adults with Progeria, one single letter in that manual is wrong – there is a “T” that should be a “C”. That single-letter change leads to the production of a toxic protein called progerin that causes the disease.

SamPro-2 is built to correct that error — permanently changing the “T” back to a “C” — with the goal of stopping the disease inside the body’s cells at its source.

Why PRF Chose Forge to Manufacture SamPro-2

Before any gene therapy can be given to a person, it must be:

• Manufactured in highly specialized, ultra-clean facilities
• Produced under strict current Good Manufacturing Practice (cGMP) standards
• Tested repeatedly for safety, purity, and consistency
• Prepared for U.S. FDA regulatory review

Forge Biologics is a leader in gene therapy manufacturing. Their expertise will help us move SamPro-2 from breakthrough laboratory science to clinical trials for children and young adults with Progeria.

Manufacturing is the bridge between discovery and treatment.

This partnership builds that bridge.

Il team del gene della progeria

PRF and its collaborators, collectively known as the Progeria Gene Team, created SamPro-2. The Progeria Gene Team brings together internationally recognized leaders in genetics, clinical medicine, and biomedical innovation, all driven by a shared commitment to finding the cure for Progeria.

Dott. Francis S. Collins, dottore in medicina e chirurgia
Senior Research Advisor to PRF and former Director of the National Institutes of Health, who led the Human Genome Project — one of the most significant scientific achievements of our time — and helped usher genetics into the modern era.

Dott. Leslie Gordon, dottore in medicina e chirurgia
PRF Co-founder and Medical Director; the world’s leading expert on Progeria and a driving force behind every major breakthrough in the field. Dr. Gordon is also the mother of Sam, whose life inspired PRF’s creation and continues to fuel its urgency.

Dott. David R. Liu
Richard Merkin Professor and Director of the Merkin Institute for Transformative Technologies in Healthcare at the Broad Institute of MIT and Harvard. Dr. Liu is a globally recognized pioneer in gene editing and an international leader in the development of base editing technologies that are reshaping modern medicine.

The late Sammy Basso, MS
Scientist, advocate, and extraordinary member of the global Progeria community. Sammy’s intellect, courage, and unwavering commitment to advancing research continue to inspire and guide this work.  Sammy was PRF’s international ambassador. Sammy and his family created Associazione Italiana Progeria Sammy Basso (A.I.Pro.Sa.B.) to help find the cure for Progeria.

Why This Is a Turning Point

For families, this is tangible momentum.

For donors, this is the impact of your investment in bold science.

For the broader rare disease community, this represents one of the most advanced efforts yet to correct a fatal, pediatric, multi-system disease at its genetic source.

We still have important work ahead. But this is a big step forward. And every step brings us closer to our vision: A world where every child with Progeria is cured.

Read the Press Release Here