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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 - 18:00
Session 1 – Exemplars of translation into clinical practice and lessons learnt
Moderators tbc
14:00 - 14:10
Welcome - Introduction - Objectives of the Workshop
To prepare a short position paper describing:
Chairpersons
14:10 - 14:30
What is precision cardiology?
Perry Elliott (London)
14:30 - 14:45
PCSK9 and beyond
Ulf Landmesser (Berlin)
14:45 - 15:00
Cardiac Amyloidosis
Marianna Fontana (London) TBC
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
15:45 - 16:00
Challenges for gene therapies: Delivery, durability and complications
Mauro Giacca (London)
16:00 - 16:15
Gene therapy and Gene editingExamples of successful translation
Christian Kupatt (Munich)
16:15 - 16:3010‘talk+10‘ disc.
RNA Therapeutics
Speaker from Alnylam
16:30-17:15
Breakout Groups & 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?
17:15 - 17:30
Break
Session 3 – DIAGNOSTICS: POLYGENIC RISK SCORES
17:30 - 17:4510‘talk+10‘ Disc
Debate: Polygenic risk scores are ready for application in routine cardiological practice.
Pro: Speaker TBC
17:45 - 18:0010‘talk+10‘ Disc
Con: Heribert Schunkert (Munich)
Panel and audience discussion “How to overcome the gaps in translation – What are the critical issues?”
18:00 – End of Day 1 - Outlook to Day 2
DAY 2: 24 March 2023 - 09:00 - 12:00
Session 3 (cont’d) – DIAGNOSTICS: POLYGENIC RISK SCORES
Challenges and opportunities - A 5’ introduction for each topic and 15’ discussion
09:00 - 09:20
What does a gene therapy trial look like?
Speaker TBC
09:20 - 09:40
What is the landscape for approval and licensing of genomic therapies (FDA, EMA, MHRA)
Antonio Gomez-Outes (Spain)
Emmanouil Zouridakis, UK MHRA
Sairah Tommy (USA) FDA
09:40 - 10:40
Breakout Groups & Feedback/discussion (3)
10:40 - 11:00
11:00 - 11:45
Wrap-up from breakout groups – Call for actions?Key take-home messages and Recommendations in preparation of a publication
12:00 – End of Day 2
Our mission: To reduce the burden of cardiovascular disease.