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Detailed Workshop Agenda
Wednesday, November 2
Check-In and Poster Set-up
 2:00-4:00PM Check-in located in the West Tower, Level Two, Foyer in front of Grand Ballroom
 2:00-4:00PM Poster Set-up:  All poster presenters (Sessions 1 and 2) are asked to set up their posters at this time in the West Tower, Level Two, Skyline A-E
Wednesday, November 2
Day 1 Kickoff Session: 4:00 PM 8:00 PM, Grand Ballroom
Dinner Included
4:00-4:30PM Introduction & Greetings:  Medical Director Leslie Gordon, MD, PhD, PRF Executive Director Audrey Gordon, Esq; Board Chair Scott Berns, MD
4:30-5:20PM In-person – Sammy Basso, Carlos  Luiz Silva, Meg Waldron
By Zoom: children from around the world
Meet the Children Around the World Living with Progeria:   Inspiring Presentations by Patient Families
5:20-5:40PM Francis Collins
National Institutes of Health, Bethesda, MD
 Progeria Musical Celebration
5:40-6:20PM Leslie Gordon
The Progeria Research Foundation, Brown University, Hasbro Children’s Hospital, Boston Children’s  Hospital, and Harvard Medical School, Boston, MA
The Lonafarnib Journey to Approval – Lessons for Future Treatments and the Cure
                                                                                                     Group Photo
 6:25PM                                                                                                      DINNER IS SERVED
Thursday, November 3
Day 2: Breakfast Buffet  / 7:25 AM – 8:25 AM, Grand Ballroom
All Posters should be set up by start of morning session.
Day 2 Morning Session
8:25 AM Progeria Clinical Trial Development Programs
Moderator:  Francis Collins, MD, PhD, National Human Genome Research Institute (NIH)
8:30-8:40AM Leslie Gordon
The Progeria Research Foundation, Brown University, Hasbro Children’s Hospital, Boston Children’s Hospital, and Harvard Medical School, Boston, MA
 Hello and Meeting Program Overview
8:40-9:10AM David Liu*
Merkin Institute of Transformative Technologies in Healthcare, Broad Institute, Howard Hughes Medical Institute, Harvard University, Boston, MA
Plenary Talk:
Base Editing and Prime Editing: Precision Gene Correction Without Double-Strand DNA Breaks

Francis Collins

David Liu

John Evans – Chief Executive Officer, Beam Therapeutics, Cambridge, MA

Leslie Gordon

Panel Presentation:
Base Editing to Rescue Progeria – where are we now, and where do we need to go?

Leslie Gordon

Francis Collins

Emma Mccullagh – Preclinical Toxicology Specialist, Domain Biotechnologies, Austin, TX

Eric Zanelli – PRF Clinical Development Volunteer Allievex Corporation, Boston MA

Panel Presentation:
Update on Progress in Development Program for SRP-2001 HGPS-specific RNA Therapy  
9:50-10:10AM Bae-Hoon Kim
PRG Science and Technology, Korea
Update on Progress in Development Program for Progerinin, Inhibitor of Progerin
10:10-10:30AM Monica Kleinman
Boston Children’s Hospital and Harvard Medical School, Boston, MA
Everolimus Plus Lonafarnib Clinical Trial Outcomes
10:30-10:50AM                                                                                                    BREAK and Group Photo
                                                                                                    Progeria Biomarkers and Progerin Turnover Across Tissues
10:50-11:10AM Abigail Buchwalter*
Cardiovascular Research Institute and the Department of Physiology at the University of California, San Francisco, CA
Combined Proteome-Wide Measurements of Protein and Cell Turnover Across Tissues Accounts for The Tissue Specificity of Progeria
11:10-11:30AM Leslie Gordon Plasma Progerin Changes with Lonafarnib    Therapy – Association with Drug Effect and Extended Survival
11:30-11:50AM Mario Cordero*
Biomedical Research and Innovation Institute of Cadiz (INIBICA), Cadiz, Spain
Biochemical Biomarker Development in Progeria

Day 2 Afternoon Session

Moderator: Maria Eriksson, PhD, Karolinska Institutet, Huddinge, Sweden

Poster talks and Lightening Poster Session 1
11:50-12:05PM Selected Poster Presentation
12:05-12:20PM Selected Poster Presentation
12:20-12:40PM Lightening Poster Session #1 – Posters #1-20 1 minute & 1 slide per poster presenter


1:40-2:00PM Lightening Poster Session  #2 – Posters #21-40 1 minute & 1 slide per poster presenter
2:00-2:20PM Maria Eriksson Single-cell RNA analysis of the murine arterial wall during HGPS disease progression
2:20-2:40PM Giovanna Lattanzi*
CNR Institute of Molecular Genetics- Bologna, Italy
Adipocyte-Specific Effects of Drug Treatment Prospects in HGPS
2:40-3:00PM John Cooke
Houston Methodist, Houston, TX
DNA Damage in Progeria, and its Reversal by Telomerase
3:00-3:20PM Late Breaking Talk
3:20-4:50PM                              Formal poster session #1: Even-Numbered Posters – Skyline A-E


6:00–8:00PM Buffet Dinner/Informal Conversation, Grand Ballroom
6:30-8:00PM Formal poster session #2: Odd-Numbered Posters – Skyline A-E
Friday, November 4
Day 3: Breakfast Buffet / 7:15 AM – 8:25 AM, Grand Ballroom
Day 3 Sunrise Session: Roundtables with Leaders In Progeria and Aging (Table Sign-up Required)
8:00-8:30AM Vicente Andrés, PhD
Sammy Basso, MSc
Francis Collins, MD, PhD
Leslie Gordon, MD, PhD
Monica Kleinman, MD
Mentors will be assigned to a table where students and junior investigators will have the ability to have significant personal exposure to senior leaders and a patient who can provide guidance and inspiration.
Day 3 Morning Session
8:40AM   Progress in Cardio-Neurovascular Treatment Outcomes in HGPS
Moderator: Marie Gerhard-Herman, MD , Brigham and Women’s Hospital, Boston MA
8:40–8:50AM Marie Gerhard-Herman Overview of Ongoing CV/NV Progress at BCH and BWH and Summary of CVIS Meeting
8:50-9:10AM Srinivas Mukundan
Reveal Pharmaceuticals, Cambridge, MA
New Stroke and Brain Vascular Natural History Findings in Progeria
9:10 -9:30AM Marie Gerhard-Herman*
Brigham and Women’s Hospital, Boston MA
Pulse Wave Velocity Portends Survival Risk in HGPS
9:30-9:50AM Pedro del Nido
Boston Children’s Hospital; Harvard Medical School, Boston, MA
TAVR and Apico-Aortic Conduit Surgery for Critical Calcific Aortic Stenosis in HGPS
9:50-10:05AM Sammy Basso
Sammy Basso’s Italian Association for Progeria, Italian Network for Laminopathies, Italy
Patient Perspective on Quality of Life 3 years Post-Aortic Valve Intervention


                                                                                                  Cardiovascular Pathobiology of Progeria
10:25-10:55AM Jay Humphrey
Department of Biomedical Engineering Yale University, New Haven, CT
Evolving Vascular Phenotypes and Cardiovascular Consequences in a Mouse Model of HGPS and Its Treatment
10:55-11:15AM Vicente Andrés*
Centro Nacional de Investigaciones Cardiovasculares (CNIC), Madrid, Spain                                  
Smooth Muscle or Endothelium: Which is to Blame for Precocious Vascular Disease and Death in HGPS?
11:15-11:35AM George Truskey
Duke University Durham, NC
Effect of Adenine Base Editing on the Function of a Tissue-Engineered Vascular Model of HGPS
11:35-11:55AM Roland Foisner
Max F. Perutz Laboratories, Medical University, Vienna, Austria
Endothelial and Systemic Upregulation of miR-34a-5p Fine-tunes Senescence in Progeria


Day 3 Afternoon Session
1:00PM New Mechanisms of Disease in Progeria
Moderator: Karima Djabali
Technical University of Munich (TUM) School of Medicine, Munich, Germany
1:00-1:20PM Chiara Lanzulo*
Institute of Biomedical Technologies (ITB)-CNR, Istituto Nazionale di Genetica Molecolare, Milan, Italy
Lessons from The Inflammasomes in the Pathophysiology of Progeria
1:20-1:40PM Valerio Orlando
King Abdullah University of Science and Technology, Kingdom of Saudi Arabia
Repeatome RNA: From Junk to Therapeutics for Premature Aging and Tissue Regeneration
1:40-2:00PM Elsa Logarinho*
Instituto de Biologia Molecular e Celular (IBMC), Porto, Portugal
Small-Molecule Correction of Microtubule-Dependent Genotoxic Stress in HGPS Cells Bypasses Progerin-Driven Cellular Phenotypes
2:00-2:20PM Karima Djabali* Modulation of JAK-STAT Signaling in Hutchinson-Gilford Progeria Syndrome
2:20-3:00PM Break and prep for Summation Session

Mark Kieran
Day One Biopharmaceuticals, Brisbane, CA

Kan Cao
University of Maryland, College Park, MD

Summation of Meeting and Key Findings

Thank you for coming!

The 2022 PRF Progeria Workshop Organizing 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

Judy Campisi, PhD – Professor, The Buck Institute; Senior Scientist, Lawrence Berkeley National Laboratory

Vicente Andrés, PhD – Professor, Laboratory of Molecular and Genetic Cardiovascular Pathophysiology, and Director Department of Basic Research, Centro Nacional de Investigaciones Cardiovasculares Carlos III (CNIC)                                                

Mark Kieran, MD, PhD – VP Clinical Development, Day One Biopharmaceuticals, a company dedicated to developing novel treatments for children

Justine Maestranzi, BA Meeting Coordinator, The Progeria Research Foundation

Joanne Morris Workshop Administrator, Joanne Morris Events