R13AG071212

The Progeria Research Foundation 10th International Scientific Workshop, "Researching Possibilities, Extending Lives" - Overview: Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) is a rare, fatal segmental premature aging disease caused by a splice site mutation in the LMNA gene. Children with Progeria die from heart attacks or strokes at an average age of 14.6 years following premature, progressive atherosclerosis.

The mission of the Progeria Research Foundation (PRF) is to find treatments and the cure for Progeria and its aging-related disorders, including cardiovascular disease (CVD). The upcoming PRF 10th International Scientific Workshop on Progeria will be held in Cambridge, MA on November 2-4, 2020. All prior PRF workshops were co-funded by the National Institutes of Health.

With record-high numbers of both peer-reviewed publications on Progeria and requests for cell lines from the PRF Cell and Tissue Bank for preclinical explorations, as well as first-ever submissions to the FDA for both drug approval and a disease biomarker, this field of study is on the verge of major advancements.

Objectives: To create an ideal environment for collaborative discussion between basic and clinical scientists about how their collective experience with Progeria can accelerate scientific progress for Progeria, CVD, and aging; to coordinate research activities; and to develop future activities in these fields towards better health for those with Progeria and for the general aging population.

Program: The meeting starts with an inspiring evening session. Progeria clinical trial patients, along with their parents, will share their experiences, feelings, and extraordinary personalities with the scientific audience. This is followed by a plenary presentation by pioneer David Liu, PhD (Broad Inst.), who will usher the attendees into the world of genetic editing and its endless possibilities for application to Progeria, CVD, and aging.

Day 2 will start with critical emerging Progeria model systems and disease biomarkers, followed by an integrated basic and clinical cardio/neurovascular panel session. Peppered throughout the program are seasoned HGPS, CVD, and aging experts, mixed with junior investigators who will undoubtedly lead this field over the coming decade.

On Day 3, an early morning "coffee talk" session will see junior investigators and students receiving mentorship from world experts. Next, data on the only active Progeria clinical trials worldwide will be presented. Future interventions then take the stage, with new data from prestigious investigators such as NIH Director Francis S. Collins, MD, PhD and NCI Distinguished Investigator Tom Misteli, PhD on RNA and DNA therapeutics, and 3 talks on post-translational pathway-based intervention, including drug repurposing.

A poignant all-hands discussion on the effects of COVID-19 on our research community and summation and key findings sessions, moderated by world-renowned aging investigator Judith Campisi, PhD, will galvanize the group with one common goal: to bring hope to those with Progeria for a long and healthy future.

Based on prior meeting trends, we estimate 200 attendees from 14 countries, 28 speakers, and 50 posters.

Conclusion: Science presented at this meeting will greatly contribute to the next wave of discovery in Progeria and its relationships to aging and CVD in the general population.

The Progeria Research Foundation

93.350 - National Center for Advancing Translational Sciences

National Institute on Aging (NIA) [HHS - NIH]

National Center for Advancing Translational Sciences (NCATS) [HHS - NIH]

Massachusetts United States

State-Wide

NIH Support for Conferences and Scientific Meetings (Parent R13 Clinical Trial Not Allowed) (PA-18-648)

Amendment Since initial award the End Date has been extended from 05/31/22 to 05/31/23 and the total obligations have increased 18% from $42,500 to $50,000.