DAY 1: THURSAY 05 MARCH 2026

 

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

Session 1: Current landscape and unmet needs in rare diseases

 

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 Topic: 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 in rare drug development.

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

Afternoon

 

Session 3: Drug discovery, development and repurposing in rare diseases

 

14:00 – 14:20 Topic: Channelling resources from national and supranational funding frameworks towards drug research and development

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

14:20 – 14:40 Topic: The role of artificial intelligence in drug discovery and repurposing

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

14:50 – 15:10 Topic: Building on knowledge and lessons learnt: 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.

Speakers:

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

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

· Anton Ussi, Coordinator of REMEDI4All

· Kaja Kantorska. Policy officer, DG SANTE

Moderator:

Dr Petros Petrou, Senior Scientist, Head of the Biochemical Genetics Department,The Cyprus Institute of Neurology & Genetics

 

16:30 Closing Remarks

 

 

 

DAY 2: FRIDAY 06 MARCH 2026

Morning

08:00 – 08:30 – Registration and coffee

 

Session 4: Becoming trial-ready for rare diseases therapies

 

08:30 – 08:45 Topic: Introducing initiatives to bring more clinical trials to Europe

Helene Le Borgne, Policy Officer, DG Research and Innovation

08:45 – 09:05 Topic: Building an ecosystem for rare disease trial readiness Maurizio Scarpa, Coordinator of MetabERN

9:05 – 9:20 Topic: The European Platform on rare disease registration: Connecting data, accelerating care pathways and enabling research

Andri Papadopoulou, Scientific Officer, Joint Research Centre

09:20 – 09:40 Topic: National Biobanks for Population and Rare Disease Research

Giedrė Kvedaravičienė, Director of the Lithuanian Population and Rare Diseases Biobank

09:40 – 10:00 Topic: Clinical trials from the patients’ perspective:

Allan Fred Byamukama

10:00 – 10:30 Panel discussion: Stakeholders working together for clinical trial preparation

Speakers:

· Helene Le Borgne, Policy Officer, DG Research and Innovation

· Maurizio Scarpa, Coordinator of MetabERN

· Virginie Hivert, Head of Therapies and Access at EURORDIS

· Andri Papadopoulou, Scientific Officer, Joint Research Centre

· Allan Fred Byamukama

· Giedrė Kvedaravičienė, Director of the Lithuanian Population and Rare Diseases Biobank

· Alexis Arzimanoglou, Coordinator of ERN EpiCARE

 

 

Moderator:

Prof. Kleopas Kleopa, Senior Neurologist, Head of the Neuroscience Department, The Cyprus Institute of Neurology & Genetics

 

 

10:30 – 11:00 Coffee Break

 

 

Session 5: Rollout of efficacious therapies: access and infrastructure considerations

 

11:00 – 11:20 – TBC

11:20 – 11:40 Topic: Health economics of expensive therapies: considerations from the State

Neophytos Charalambides, Minister of Health, Republic of Cyprus

11:40 – 12:00 Topic: Infrastructure considerations for delivering therapies at scale:

 

Anne-Sophie Lapointe, French representative in ERN Board of Member States

12:00 – 12:20 Topic: Health Technology Assessment and Innovative Payment Models for therapies for rare diseases

Christine Leopold, Pharmaceutical system and policy researcher, Utrecht Center for Pharmaceutical Policy and Regulation / WHO Collaborating Center

12:20 – 12:40 – Topic: Competitive (mis)pricing in small markets: an example of DMD therapies

Panikos Voskos, President MDA Cyprus

12:40 – 13:10 – Panel discussion: Rare disease therapy delivery in the healthcare system: paths to sustainability.

Speakers:

· Neophytos Charalambides, Minister of Health, Republic of Cyprus

· Anne-Sophie Lapointe, French representative in ERN Board of Member States

· Christine Leopold, Pharmaceutical system and policy researcher, Utrecht Center for Pharmaceutical Policy and Regulation / WHO Collaborating Center

· Panikos Voskos, President MDA Cyprus

· Stella Kyriakides, former EC Health Commissioner

Moderator:

Dr Menelaos Pipis, Senior Neurologist, Head of the Neuropathology Department, The Cyprus Institute of Neurology & Genetics

 

 

13:10 Closing Lecture

The role of CING in caring for patients with rare diseases: clinical excellence and research innovation

Leonidas Phylactou, CEO & Medical Director, CING

 

End of day 2 – 13:30