PRF’s Journey to the Cure Featured in New Yorker Magazine
The Path to Cure Progeria is Here…
After 6 years of intense work, culminating in the final design of the gene therapy drug that could cure Progeria, PRF’s pioneering Gene Editing program is ramping up its next stage of development: manufacturing the drug for human clinical trials.
Base editing gene therapy uses CRISPR technology to target a specific DNA base (or DNA letter) and change it to another. Because Progeria is caused by a change to a single letter in the DNA, it is perfectly suited for this type of gene therapy.
The “Gene Dream Team”!
PRF has been working non-stop with an extraordinary team* of world-class innovators to bring this potentially transformative therapy to reality, including:
- Francis Collins, MD PhD, longtime Progeria research partner and former Director of the National Institutes of Health
- David Liu, PhD, Broad Institute of MIT and Harvard University, and developer of Base Editing Gene Therapy Technology
- Michael Erdos, PhD Associate Investigator in the Molecular Genetics Section of the Center for Precision Health Research, National Human Genome Research Institute (NHGRI)
- Leslie Gordon, MD PhD, PRF Co-founder & Medical Director, and the world’s expert on Progeria
*Sammy Basso, MS, Co-Founder of The Associazione Italiana Progeria Sammy Basso and PRF Ambassador, was an integral part of this team until his passing in October 2024.
The Progeria Gene Team started by showing that gene editing could increase the lifespan of the Progeria mouse model (by 140%, which they published in the world’s top scientific journal, Nature. After that incredible accomplishment, the team worked to refine the drug. is now working on drug manufacturing, US FDA interactions, and clinical trial development to bring this promising technology to children and young adults with Progeria. It’s a multi-stage, technically complicated, expensive, and time-consuming process – and PRF is up to the challenge.
The Progeria Community has been following along with the Gene Team’s progress from the beginning. PRF’s leadership team conducts regular Zoom meetings with Progeria families throughout the world, so they can be our partners in this journey towards the cure!
Moreover, the success of this project could have widespread implications for millions of others with rare genetic diseases.
“If gene editing is effective in slowing or halting progeria, the methods may also help or treat other rare genetic diseases that have no treatments or cures and, like progeria, have aroused little interest from drug companies.” – New York Times, Disease That Makes Children Age Rapidly Gets Closer to a Cure. July, 2024
♦ In addition to curing Progeria, we are forging a path for others to take – how incredibly exciting!
To bring Progeria gene editing therapy to the clinical trial stage will require many millions of dollars as we enter the next stages of this program. As it has been from the beginning, it is up to PRF and its dedicated supporters to continue this promising, innovative work to cure Progeria. With your continued support, Progeria Gene Therapy is now within our sights!
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