Thursday 22 June
Session 1: Introduction
Session 2: Gene therapy: from technology to clinical trials
14:00
Welcome and introduction
Robin Lachmann, Stefan Kölker, Alastair Kent
14:15
10 years away: A brief history of gene therapy
Paul Gissen, UK
14:45
Highs and lows: The patient perspective
Alastair Kent, UK
15:15
The modern gene therapy toolbox
Alessandro Aiuti, Italy
15:45
Delivery and expression; the vector and the cargo
Simon Waddington, UK
16:45
Ex-vivo gene therapy for neurometabolic diseases
Alessandro Aiuti, Italy
17:15
Liver-directed gene therapy for IMDs
TBC
17:45
Gene therapies of the hematopoietic system - challenges and opportunities
Christoph Klein, Germany
18:15
Q&A
Friday 23 June
Session 3: RNA therapy plus: on sense and antisense
Session 4: Clinical trial design
Session 5: Workshops: you design the study
09:00
Delivering the message: Principles of mRNA therapy in IMDs
TBC
09:30
Interfering with the message: Antisense oligonucleotide therapies for neurological disease
Marlen C. Lauffer, The Netherlands
10:00
Coming soon – Gene editing technologies
TBC
11:00
Withdrawing therapy; how and when do we stop our current treatment in genetic therapy trials?
Derralyn Hughes, UK
11:30
How do we measure the lifetime efficacy of a one-off treatment?
TBC
12:00
How do we involve the patients most in need of treatment?
Paul Gissen, UK
13:30-18:30
Interactive workshops: You design the study
Workshop 1: The Enzyme factory
A ‘gene therapy’ for an LSD
Derralynn Hughes, Simon Waddington
Workshop 2: Reaching the brain
Local vs global gene delivery
Alessandro Aiuti, TBC
Workshop 3: Adjusting the dose
Liver directed mRNA therapy
Charles Venditi, Robin Lachmann
18:30
Discussion of workshop results
Saturday 24 June
Session 6: Ensuring access
Session 7: Ethics and patient perspective
08:30
Selling a cure: the pricing of one-off treatments for rare diseases
Mondher Toumi, France
09:00
Value for money in rare disease
Amanda Whittal, Germany
09:30
Paying models: Pay upfront or pay as you go?
Andreas Ziegler, Germany
10:00
Decision making on patient access to gene therapies
Ron Akehurst, UK
11:00
Panel discussion: Ethics and patient perspective
Alastair Kent, Derralyn Hughes, Paul Gissen, Mondher Toumi