Detailed Workshop Agenda |
||
Wednesday, November 2
|
||
| 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
|
||
| 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 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 |
| 9:10-9:30AM |
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? |
| 9:30-9:50AM |
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 |
| 12:40-1:40PM |
LUNCH |
|
| 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 | |
|
ADJOURN UNTIL DINNER AND EVENING SESSION |
||
| 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 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 |
|
10:05-10:25AM |
BREAK | |
| 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 |
| 12:00-1:00PM |
LUNCH |
|
| 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 | |
| 3:00-3:55PM |
Mark Kieran Kan Cao |
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
Sign Up
for Our
Newsletter!
Together, we
WILL
find the cure!
