The Progeria Research Foundation 12th Annual International Scientific Workshop: Coming of Age - Progeria Research Reaching for the Cure

Progeria Research Foundation, Inc., Peabody MA

Investigators

Abstract

The Progeria Research Foundation 12th Annual International Scientific Workshop: Coming of Age – Progeria Research Reaching for the Cure Abstract Overview: Hutchinson-Gilford progeria syndrome (HGPS or Progeria) is an ultrarare, fatal, genetic premature aging disease caused by progerin, a toxic protein generated by a splice site-generating mutation in the LMNA gene. Children and young adults die from heart failure that is consequent to premature, progressive atherosclerosis. The Progeria Research Foundation’s (PRF’s) mission is to find treatments and the cure for Progeria and its aging-related disorders, including cardiovascular disease (CVD). PRF’s 12th International Scientific Workshop on Progeria will be held in Cambridge, MA on October 29-31, 2025. All prior PRF workshops were co-funded by the National Institutes of Health, a strong partner in aging and rare disease research. As more children with Progeria live to their late teens and early 20s due to Progeria’s first FDA-approved treatment, the scientific and patient communities are ready to galvanize around achieving even healthier, longer lives for these patients through translational research. Objectives: to create an ideal environment for collaborative interactions between basic and clinical scientists around how their collective experience can accelerate research for Progeria, CVD and aging; to foster young scientists who represent the future of Progeria research; to support a diverse Progeria community; and to plan future activities towards better health and longer lifespans for those with Progeria and for the general aging population. Program: The opening evening session will be led by 5 young adults with Progeria whose lives began with no identified genetic cause, no treatments, and no hope, but who now have all these things. They’ll share their lifelong journeys as partners in Progeria research success. Day 2 starts with high translational impact. Drs. Monica Kleinman and Leslie Gordon will report on new clinical trials (the only active Progeria trials in the world) using progerin-targeted small molecules. This is followed by data- rich basic and clinical cardiovascular presentations that, together, will help us understand which cell types should be priority targets for treatment. Finally, junior investigators take the stage for lightening round, 1-minute introductions to the posters they will present during the evening session. Day 3 starts with a sunrise coffee hour where patients and their families will sit at various tables and inspire meeting attendees in a small group setting. Future interventions then take the stage, with 4 upstream treatment strategies in development: 1 progerin- targeted small molecule, 2 classes of RNA-based therapy, and finally, precise base editing that could one day permanently correct the LMNA mutations and cure Progeria. Gene therapy pioneer Dr. David Liu and former NIH Director Dr. Francis S. Collins have teamed up with PRF to bring in vivo HGPS gene correction to the clinic. The workshop wraps up with a summary session where our newly invigorated “Progeria research family” shares key learnings and creates a Progeria-aging-CVD action plan. We estimate 200 attendees and 20-30 posters. Conclusion: Collegial interactions along with outstanding science presented at this meeting will generate the next wave of translational discovery for Progeria, atherosclerosis of aging.