Summary
A workshop chaired by Profs. Stefanie Dimmeler and Perry Elliott, Dr. Leticia Ferri (BMS), Dr. Paul Nioi (Alnylam) and Dr. Chris O’Donnell (Novartis).
A workshop chaired by Profs. Stefanie Dimmeler and Perry Elliott, Dr. Leticia Ferri (BMS), Dr. Paul Nioi (Alnylam) and Dr. Chris O’Donnell (Novartis).
DAY 1: 23 March 2023 - 14:00 - 18:45
Time | Session | Speaker |
Session 1 – Exemplars of translation into clinical practice and lessons learnt Chaired by Chris O'Donnell and Heribert Schunkert |
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14:00 - 14:10 |
Welcome - Introduction - Objectives of the Workshop To prepare a short position paper describing:
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Chairpersons |
14:10 - 14:30
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Perry Elliott (London) |
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14:30 - 14:45 |
PCSK9 and beyond |
Ulf Landmesser (Berlin) |
14:45 - 15:00 |
Thibaut Damy (Paris) |
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15:00- 15:45 |
Breakout Groups & Feedback/discussion (1) What does success look like? |
Moderators |
Session 2 – CURRENT STATE OF THE ART: CHALLENGES FOR GENE THERAPY Chaired by Filippo Crea and Paul Nioi |
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15:45 - 16:00 |
Challenges for gene therapies: Delivery, durability and complications |
Mauro Giacca (London) |
16:00 - 16:15 |
Gene therapy and Gene editing
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Christian Kupatt (Munich)
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16:15 - 16:30 |
Weinong Guo (Alnylam)
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16:30-17:15 |
Breakout Groups and Feedback/discussion (2) 1. What are the main technical/biological barriers to be overcome? 2. What can we do collectively to accelerate translation to patients?
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Moderators |
17:15 - 17:30 |
Break
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Session 3 – DIAGNOSTICS: POLYGENIC RISK SCORES Chaired by Stefanie Dimmeler and Perry Elliott |
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17:30 - 17:45 |
Debate: Polygenic risk scores are ready for application in routine cardiological practice.
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Pro: Maryam Kavousi (Rotterdam) |
17:45 - 18:00 |
Debate: Polygenic risk scores are ready for application in routine cardiological practice.
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Con: Heribert Schunkert (Munich)
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18:00 - 18:45 |
Panel and audience discussion “How to overcome the gaps in translation – What are the critical issues?” |
All |
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Short rebuttal |
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VOTE BY AUDIENCE FOR/AGAINST THE MOTION |
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18:45 – End of Day 1 - Outlook to Day 2 |
DAY 2: 24 March 2023 - 09:00 - 12:00
Time | Session | Speaker |
Session 3 (cont’d) – DIAGNOSTICS: POLYGENIC RISK SCORES Chaired by Leticia Ferri and Eva Prescott |
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09:00 - 09:15 |
Summary of Day 1 – Main outcomes from the breakout discussions |
Chairpersons |
09:15 - 09:45 |
What is the landscape for approval and licensing of genomic therapies (EMA, MHRA) |
Antonio Gomez-Outes (Spain) Emmanouil Zouridakis, UK MHRA |
09:45 - 10:45 |
Breakout Groups and Feedback/discussion (3)
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Moderatored by the chairpersons |
10:45 - 11:00 |
Break |
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11:00 - 11:45 |
Wrap-up from breakout groups – Call for actions? |
Chairpersons |
12:00 – End of Day 2 |