Challenges and barriers for gene therapies in rare neurological disorders: preparing the next generation of clinicians and researchers

Concept
Advances in gene therapies are completely changing the possibilities we used to have to deal with devastating rare neurological disorders and dramatically changing our expectations regarding outcomes in these patients. More than 50 gene therapies could be in the clinical ground in the next 10 years, but there are still lots of uncertainties and challenges we need to cope with.

Aim
The aim of this course is to discuss known barriers, challenges, and uncertainties in gene replacement therapies including the vision of different stakeholders (basic researchers, clinicians, patients, regulators, payers, and industry. We split the course into two days in which we deal with different bottlenecks. On the first day, we will review the state of the art in therapy development and problems of the current viral carriers, and possible solutions. On the second day, we will discuss the problems related to clinical implementation and safety.

The course is open for clinicians, clinical researchers and translational researchers who are or want to be involved in the implementation and development of genetic therapies in the 2025-2035 period.

Hybrid (face-to-face or online)

To register please fill the application form here until May 31, 2023. 

The training workshop is free of charge.
Travel and hotel expenses will be reimbursed for all selected ERN participants (max 20). Lunch and dinner will be provided on site for all participants.

Affiliated to an ERN-Full Member or Affiliated Partner institution.

The organizer together with one member of the WP17 Scientific Evaluation Committee will select the most appropriate workshop participants based on participation in ERN activities, age (those who will be leading this in Europe in 10 years to come) and demonstration of previous involvement in gene therapy.  Priority will be also given to authors of case study posters.

All registered participants will be informed if they are selected to attend this training course before 06 June 2023.  

At the end of the training workshop a Certificate of Attendance will be sent to the participants who attended the entire workshop and completed the survey. 

English.

Hospital Universitari Vall d’Hebron (Barcelona)

If you have specific questions, please write to the workshop organizer:
David Gómez-Andrés (david.gomezandres@vallhebron.cat)

For general inquiries about the workshops in the ERN RD Support and Training Program please write to:
Carola.Reinhard@med.uni-tuebingen.de
c.n.van_beuzekom@lumc.nl

See the programme here.