Agenda | The Progeria Research Foundation

Agenda for The Progeria Research Foundation International Scientific Workshop

Agenda at a Glance Note: Agenda Subject to Change
Wednesday, October 29, 2025
Time	Session	Speaker
3:00-5:00 PM	Registration & Poster Set-Up
5:00-5:15 PM	Introduction & Greetings from PRF	Audrey Gordon, Esq. Executive Director The Progeria Research Foundation Scott Berns, MD, MPH, FAAP Board Chair The Progeria Research Foundation Leslie Gordon, MD, PhD Medical Director The Progeria Research Foundation
5:15-5:20 PM	PRF Medical Research Remarks	Leslie Gordon
5:20-6:20 PM	“Coming of Age” Conversations: Young Adults with HGPS as Research Partners	Moderator: Merlin Waldron Kaylee Halko Amber Vandeweert-Wielemans Michiel Vandeweert Riccardo Zanolli
6:20-6:30 PM	Musical Kickoff	Francis Collins, MD, PhD
6:30 PM	Dinner & Networking

Thursday, October 30, 2025
7:15 AM-8:30 PM	Registration Desk Open
7:30-8:30 AM	Bridging the Bench Mentor Session
8:30-9:00 AM	Breakfast
Current Treatment Trial Outcomes in Progeria Moderator: Mark Kieran, MD, PhD, VP Clinical Development, Day One Biopharmaceuticals
9:00-9:20 AM	Long-term Natural History Lonafarnib Treatment Trial	Leslie Gordon, MD, PhD
9:25-9:45 AM	Phase 2a Progerin in Clinical Treatment Trial for Progeria	Monica Kleinman, MD Boston Children’s Hospital Harvard Medical School
9:50-10:10 AM	Tools for Pivotal Science and Medicine in Progeria: Biomarker Assay Development, Qualification and Reagent Generation	Barbara Natke, PhD Chief Operating Officer and Biomarker Lead The Progeria Research Foundation
10:15-10:35 AM	Late-Stage Treatment of Aortic Stenosis in HGPS	Ashwin Prakash, MD Pediatric Cardiologist Boston Children’s Hospital
10:40-11:00 AM	Break
11:05-11:25 AM	Talk to be Announced
11:30 AM-12:00 PM	Poster Lightning Round	Poster Presenters
12:00-1:30 PM	Lunch and Networking
Cardiovascular Disease in HGPS and Aging Moderator: Maria Eriksson, PhD, Professor, Karolinska Institute
1:30 -1:50 PM	New Pathological Studies Reveal HGPS Pathophysiology	Andrea Porzionato, PhD Professor University of Padova
1:55-2:15 PM	Single-cell Analysis Reveals the Phenotypic Landscape of Arterial Cells in Progeria	Maria Eriksson, PhD
2:20-2:40 PM	Title To Be Announced	Keri Shafer, MD UT Southwestern Medical Center Sheila Hegde, MD Brigham and Women’s Hospital
2:45-3:05 PM	Angiopoietin-2: A Potential Therapeutic Target for Vascular Protection in Hutchinson-Gilford Progeria Syndrome	Kan Cao, PhD Professor and Associate Chair Department of Cell Biology and Molecular Genetics University of Maryland
3:05-3:25 PM	Break
3:25-3:45 PM	Evaluation of Coronary Artery and Aortic Calcium by Computed Tomography in Progeria	Sunil Ghelani, MD Attending Cardiologist Boston Children’s Hospital
3:50-4:10 PM	Progeria and Vascular Calcification: Diet and Treatments	Ricardo Villa Bellosta, PhD Group Leader, Ramon y Cajal Researcher Center for Research in Molecular Medicine and Chronic Diseases
4:15-4:25 PM	Poster Elevation – To be Announced
4:25-4:35 PM	Poster Elevation – To be Announced
4:35 -5:30 PM	Break
5:30-6:30 PM	Poster Session Part 1	Even Numbered Posters
6:30-7:30 PM	Dinner & Networking*
7:30-8:30 PM	Poster Session Part 2	Odd Numbered Posters

Friday, October 31, 2025
7:30 AM-2:00 PM	Registration Desk Open
7:30-8:30 AM	Breakfast & Networking
Progeria Treatment Discovery for a Healthy Future Moderator: Leslie Gordon
8:30-8:50 AM	AI-driven Therapeutic Antisense Oligonucleotide for HGPS	Ning Shen, PhD Professor Liangzhu Laboratory, Zhejiang University School of Medicine
8:55-9:15 AM	Development of Gene Silencing (RNAi) and Gene Editing Therapeutics for Hutchinson-Gilford Progeria Syndrome (HGPS)	Nizar Saad, PhD Abigail Wexner Research Institute, Nationwide Children’s Hospital
9:20-9:45 AM	Mini Plenary: Cutting Edge Translational Gene Therapy	David Liu, PhD Thomas Dudley Cabot Professor of the Natural Sciences Howard Hughes Medical Institute Investigator Harvard University Broad Institute
9:50-10:10 AM	ProSPER: PROgeria Streamlined Platform Gene Editing for Rare diseases	David Liu Francis Collins Leslie Gordon Mike Erdos, PhD Associate Investigator National Human Genome Research Institute
10:15-10:35 AM	First-Generation PROTACs for Improving Progeria Phenotype	Silvia Ortega Gutierrez, PhD Professor Universidad Complutense de Madrid
10:35-10:55 AM	Break
Endocrine and GI Disease in Progeria Moderator: Vicente Andrés, PhD, Professor, Centro Nacional de Investigaciones Cardiovasculares Carlos III
10:55-11:15 AM	Lipodistrophy and Cardiovascular Pathology in HGPS	Vicente Andrés
11:20-11:40 AM	Unraveling Accelerated Intestinal Ageing in HGPS Physiopathology: An Integrative Approach	Laurence Arbibe, MD, PhD Director INSERM Institut Necker Enfants Malades
11:20-11:40 AM	First-Generation PROTACs for Improving Progeria Phenotype	Silvia Ortega Gutierrez, PhD
11:45-12:05 PM	Targeting Inflammation to Ameliorate HGPS Pathologies	Susana Gonzalo, PhD Professor St. Louis University School of Medicine
12:10-12:50 PM	Workshop Summary Session All Hands Discussion*
12:50 PM	Lunch & Networking

2025 PRF International Scientific Workshop Scientific Committee

Leslie B. Gordon, MD, PhD (Chair) – Medical Director, The Progeria Research Foundation; Professor of Pediatrics, Hasbro Children’s Hospital, Boston Children’s Hospital, and Harvard Medical School

Vicente Andres, PhD – Professor, Centro Nacional de Investigaciones Cardiovasculares Carlos III

Maria Eriksson, PhD – Professor, Karolinska Institute

Mark Kieran, MD, PhD – VP Clinical Development, Day One Biopharmaceuticals

2025 PRF International Scientific Workshop Planning Committee

Leslie B. Gordon, MD, PhD (Chair) – Medical Director, The Progeria Research Foundation; Professor of Pediatrics, Hasbro Children’s Hospital, Boston Children’s Hospital, and Harvard Medical School

Audrey Gordon, Esq – President and Executive Director, The Progeria Research Foundation

Gina Incrovato – Director of Operations, The Progeria Research Foundation

Darrien Marazzo – Clinical Trial Coordinator, The Progeria Research Foundation

Karen Gordon Betournay– Social Media and Website Coordinator, The Progeria Research Foundation

Elizabeth Maynard – Elizabeth Maynard, CMP, Meeting Planner, Etre Partners

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Agenda at a Glance

Note: Agenda Subject to Change

Wednesday, October 29, 2025

Thursday, October 30, 2025

Friday, October 31, 2025