Session 1 – Exemplars of translation into clinical practice and lessons learnt

 Chaired by Chris O'Donnell and Heribert Schunkert

14:00 -  14:10

Welcome - Introduction - Objectives of the Workshop

To prepare a short position paper describing:

1. Current state of the art in genetic diagnostics and therapy.
2. Describe the current and future barriers to translation.
3. Propose an action plan to accelerate into routine care.                                                                               

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

Thibaut Damy (Paris) 

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

15:45 - 16:00

Challenges for gene therapies: Delivery, durability and complications

Mauro Giacca (London)

16:00 - 16:15

Gene therapy and Gene editing
Examples of successful translation

Christian Kupatt (Munich)

16:15 - 16:30

RNA Therapeutics

Weinong Guo (Alnylam) 

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?

Moderators

17:15 - 17:30

Break

Session 3 – DIAGNOSTICS: POLYGENIC RISK SCORES

Chaired by Stefanie Dimmeler and Perry Elliott

17:30 - 17:45

Debate: Polygenic risk scores are ready for application in routine cardiological practice.

Pro: Maryam Kavousi (Rotterdam) 

17:45 - 18:00

Debate: Polygenic risk scores are ready for application in routine cardiological practice.

Con: Heribert Schunkert (Munich)

 18:00 - 18:45

Panel and audience discussion “How to overcome the gaps in translation – What are the critical issues?”

 All 

Short rebuttal

VOTE BY AUDIENCE FOR/AGAINST THE MOTION

18:45 – End of Day  1 - Outlook to Day 2

Session 3 (cont’d) – DIAGNOSTICS: POLYGENIC RISK SCORES

Chaired by Leticia Ferri and Eva Prescott 

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)

E. ZOURIDAKIS - Regulatory aspects-Genomic therapies

Antonio Gomez-Outes (Spain)

Emmanouil Zouridakis, UK MHRA

09:45 - 10:45

Breakout Groups and Feedback/discussion (3)

1. How do we prioritize diseases for gene therapy?
2. What are meaningful end-points in gene trials
3. What should professional societies/organizations be doing to prepare for precision diagnostics and therapy in CVD?

Moderatored by the chairpersons 

10:45 - 11:00

Break

11:00 - 11:45

Wrap-up from breakout groups – Call for actions?
Key take-home messages and
Recommendations in preparation of a publication

Chairpersons

12:00 – End of Day  2