08:00- 08:30
Registration and coffee
08:30- 08:35
Opening and welcome Address
Welcome Address and endorsement
08:35- 08:40
Welcome Address and endorsement
(pre-recorded video)
08:40- 08:50
Welcome Address
08:50-09:00
Overview of the Rare Disease Landscape in Cyprus
Session 1: Current landscape and unmet needs in rare diseases
09:00- 09:05
Pre-recorded video
09:05-09:20
European research on rare diseases: a long-standing commitment to make Europe a leader at global level
09:20-09:35
Witnessing and implementing change over the past 2 decades: the European Commission’s action on rare diseases and therapy development
09:35-09:45
The European Economic and Social Committee’s exploratory opinion on the advancement of treatments for Rare Diseases
09:45-10:00
Regulatory considerations for rare disease drug development and approval
10:00- 10:15
EURORDIS’ role in facilitating therapies for rare diseases (online)
10:15 – 10:45
Panel discussion: Creating a Union of Opportunity for rare diseases therapy development
10:45- 11:15
Coffee Break
Session 2: A framework for developing rationally-designed therapies for rare diseases
11:15- 11:30
Funding and patient advocacy organisations spearheading the effort against rare diseases
11:30- 11:45
A European inventory of rare diseases through the scope of ERNs
11:45- 12:00
The current state of affairs for developing rational therapies for rare diseases
12:00 – 12:15
ERDERA research initiatives for therapy development in rare diseases
12:15 – 12:25
European Networking for Rare: Equity or Justice?
12:25 - 13:15
Panel discussion: Creating incentives and overcoming bottlenecks in rare drug development
13:15-14:15
Lunch break
Session 3: Drug discovery, development and repurposing in rare diseases
14:15- 14:30
Building on knowledge and lessons learnt: developing Nusinersen for SMA (online)
14:30 – 14:45
Developing virally-delivered genetic therapies for inherited neuromuscular disorders
14:45- 15:00
A Journey to Developing Curative Therapies for Hemoglobinopathies using Genome Editing
15:00 – 15:15
Developing therapies for rare paediatric endocrine disorders
15:15-15:30
Coffee Break
15:30-15:45
Chasing Miracles of Science: Accelerating Innovation in Rare Disease Medicines
15:45-16:00
The role of artificial intelligence in drug discovery and repurposing
16:00-16:10
The End of Medicine As We Know It
16:10-16:20
Innovative platform-based approach for sustainable repurposing of medicines for rare diseases
16:20-16:30
A clinical model for interdisciplinary sharing of knowledge and innovation for the development of rare diseases therapies (online)
16:30-17:30
Panel discussion: Synergies in academia and industry to accelerate drug development