Day 1

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

Leonidas Phylactou, CEO & Medical Director, CING

Welcome Address and endorsement

Prof. Michael Komodromos, President of the Board of Directors of The Cyprus Institute of Neurology & Genetics

08:35- 08:40
Welcome Address and endorsement

Olivér Várhelyi, EC Health and Animal Welfare Commissioner (pre-recorded video)

08:40- 08:50
Welcome Address

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

08:50-09:00
Statement on Rare Diseases in Cyprus (Topic TBC)

Mr. Neophytos Charalambides, Minister of Health of the Republic of Cyprus

Session 1: Current landscape and unmet needs in rare diseases

09:00- 09:15
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

09:15- 09:30
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

09:30- 09:45
Topic: The European Economic and Social Committee’ exploratory opinion on the advancement of treatments for Rare Diseases

Dr. Milena Angelova, Member at the European Economic and Social Committee; rapporteur of the opinion

09:45- 10:00
Topic: Regulatory considerations for rare disease drug development and approval

Tim Leest, Chair of EMA Committee for Orphan Medicinal Products

10:00- 10:15
Topic: EURORDIS’ role in facilitating therapies for rare diseases

Virginie Hivert, Acting CEO and Head of Therapies and Access at EURORDIS

10:15 – 10:45
Panel discussion: Creating a Union of Opportunity for rare diseases therapy development

Speakers:

  • Carmen Laplaza Santos, Head of Unit Health Innovations and Ecosystems, DG Research & Innovation
  • Stella Kyriakides, former EC Health Commissioner
  • Dr. Milena Angelova, Member at the European Economic and Social Committee; rapporteur of the opinion
  • Francois Lamy, Vice President of AFM Telethon France
  • Virginie Hivert, Acting CEO and Head of Therapies and Access at EURORDIS

 

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

10:45- 11:15
Coffee Break

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

11:15- 11:30
Topic: Funding and patient advocacy organisations spearheading the effort against rare disease

Francois Lamy, Vice President of AFM Telethon France

11:30- 11:45
Topic: TA European inventory of rare diseases through the scope of ERNs

Donata Meroni, European Commission DG Sante

11:45- 12:00
Topic: The current state of affairs for developing rational therapies for rare diseases

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

12:00 – 12:15
ERDERA research initiatives for therapy development in rare diseases

Dr. Daria Julkowska, Scientific coordinator of ERDERA

12:15 – 12:30
Topic TBC

Dr. Violetta Anastasiadou, Chair of Cyprus National Committee for Rare Diseases

12:30- 13:15
Panel discussion: Creating incentives and overcoming bottlenecks in rare drug development

Speakers:

  • Tim Leest, Chair of EMA Committee for Orphan Medicinal Products
  • Donata Meroni, European Commission DG Sante
  • Dr. Holm Graessner, Lancet Commissioner on Rare Diseases and ERN-RND coordinator
  • Dr. Daria Julkowska, Scientific coordinator of ERDERA
  • Dr. Violetta Anastasiadou, Chair of Cyprus National Committee for Rare Diseases
  • Kaja Kantorska, Policy officer, DG SANTE

 

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

13:15-14:15
Lunch break

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

14:15- 14:30
Topic: Building on knowledge and lessons learnt: developing Nusinersen for SMA

Dr. Priya Singhal, Head of Development, Biogen

14:30 – 14:45
Topic: Developing virally-delivered genetic therapies for inherited neuromuscular disorders

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

14:45- 15:00
Topic: A Journey to Developing Curative Therapies for Hemoglobinopathies using Genome Editing

Dr. Matthew H. Porteus, Stanford University School of Medicine, Stanford, USA

15:00 – 15:15
Developing therapies for rare paediatric endocrine disorders

Dr. Nicolas Nicolaides, University of Cyprus

15:15-15:30
Break
15:30-15:45
TBC
15:45-16:00
Topic: The role of artificial intelligence in drug discovery and repurposing

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

16:00-16:10
Innovative platform-based approach for sustainable repurposing of medicines for rare diseases

Harald Schmidt, Coordinator of REPO4EU

16:10-16:20
Topic: Innovative platform-based approach for sustainable repurposing of medicines for rare diseases

Anton Ussi, Coordinator of REMEDI4All

16:20-16:30
Topic: A clinical model for interdisciplinary sharing of knowledge and innovation for the development of rare diseases therapies

Prof. Birute Tumiene, Assoc Prof. at Vilnius University, Faculty of Medicine (online)

16:30-17:30
Panel discussion: Synergies in academia and industry to accelerate drug development

Speakers:

  • Dr. Priya Singhal, Head of Development, Biogen
  • Dr. Kleopas Kleopa, Senior Neurologist, Head of the Neurosciences Department, The Cyprus Institute of Neurology & Genetics
  • Dr. Matthew H. Porteus, Stanford University School of Medicine, Stanford, USA
  • Dr. Nicolas Nicolaides, University of Cyprus
  • Demetris Skourides, Cyprus Chief Scientist for Research, Innovation and Technology
  • Anton Ussi, Coordinator of REMEDI4All

 

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