Day 1

08:00- 08:30
Registration and coffee
08:30- 08:35
Opening and welcome Address

Leonidas Phylactou, CEO & Medical Director, CING

08:35- 08:40
Welcome Address and endorsement

Ekaterina Zaharieva, Commissioner for Startups, Research & Innovation

08:40- 08:45
Welcome Address and endorsement

Olivér Várhelyi, EC Health and Animal Welfare Commissioner

08:45- 08:55
Welcome Address

H.E. Mr. Nikos Christodoulides, President of the Republic of Cyprus

09:00- 09:20
Topic: European research on rare diseases: a long-standing commitment to make Europe a leader at global level

Carmen Laplaza Santos, Head of Unit Health Innovations and Ecosystems, DG Research & Innovation

09:20- 09:40
Topic: Witnessing and implementing change over the past 2 decades: the European Commission’s action on rare diseases and therapy development

Stella Kyriakides, former EC Health Commissioner

09:40- 10:00
Topic: A European inventory of rare diseases through the scope of ERNs

Donata Meroni, European Commission DG SANTE

10:00- 10:20
Topic: Funding and patient advocacy organisations spearheading the effort against rare disease

Francois Lamy, Vice President of AFM Telethon

10:20- 10:40
EURORDIS’ role in facilitating therapies for rare diseases

Virginie Hivert, Head of Therapies and Access at EURORDIS

10:40- 11:10
Coffee Break

Session 2: A framework for developing rationally-designed therapies for rare diseases

11:10- 11:30
Topic: Regulatory considerations for rare disease drug development and approval

Tim Leest, Chair of EMA Committee for Orphan Medicinal Products

11:30- 11:50
Topic: The current state of affairs for developing rational therapies for rare diseases

Holm Graessner, Lancet Commissioner on Rare Diseases and ERN‑RND Coordinator

11:50- 12:10
Topic: ERDERA research initiatives for therapy development in rare diseases

Daria Julkowska, Scientific Coordinator of ERDERA

12:10- 13:00
Panel discussion: Creating incentives and overcoming bottlenecks

Speakers:

· Tim Leest, Chair of EMA Committee for Orphan Medicinal Products

· Holm Graessner, Lancet Commissioner on Rare Diseases and ERN-RND coordinator

· Daria Julkowska, Scientific coordinator of ERDERA

· Donata Meroni, European Commission DG Sante

· Francois Lamy, Vice President of AFM Telethon

· Violetta Anastasiadou, Chair of Cyprus National Committee for Rare Diseases

Moderator:

Dr Carsten Lederer, Senior Scientist, Head of the Blood Disorders Genetics and Thalassaemia Department,The Cyprus Institute of Neurology & Genetics

13:00- 14:00
Lunch break
14:00- 14:20
Channelling resources towards drug R&D

Nicodemos Damianou, Deputy Minister of Research, Innovation and Digital Policy, Republic of Cyprus

14:20- 14:40
The role of artificial intelligence in drug discovery

Demetris Skourides, Cyprus Chief Scientist for Research, Innovation and Technology

14:50- 15:10
Developing Nusinersen for SMA
15:10- 15:30
Topic: Innovative platform-based approach for sustainable repurposing of medicines for rare diseases

Anton Ussi, Coordinator of REMEDI4All

15:30- 15:50
Topic: The success story of CRISPR gene editing in sickle cell and thalassemia
15:50- 16:30
Panel discussion: Synergies in academia and industry to accelerate drug development

Moderator: Dr Petros Petrou

16:30- 16:40
Closing Remarks