ACADEMY PROGRAMME
Check the programme regularly for updates
Wednesday 14 January
11:30-12:00
Welcome
Introduction round and selection of the order of the student presentation
12:00-12:45
Lunch
12:45-13:30
Hildegard Büning, Hannover Medical School
Adeno-Associated Virus (AAV) vectors as delivery tools in neuromuscular diseases
​​​​​13:30-14:00
Capucine Trollet, Institut de Myologie
Cell and Gene Therapy Approaches for Oculopharyngeal Muscular Dystrophy
​
14:00-15:30 Student talks 1, 2
15:30-16:00
Coffee Break​
16:00-16:30
Mario Amendola, Genethon
Genome Editing and AAV Delivery for Utrophin-mediated Duchenne Muscular Dystrophy Therapy
​
16:30-18:00 Student talks 4
18:30
Surprise Networking evening
Thursday 15 January
09:00-09:30
Isabelle Richard, Genethon
Therapeutic Challenges in Muscular Dystrophies
09:30-10:00
Federic Relaix, UPEC Health Faculty, INSERM, ENVA, EFS & APHP
Rat Duchenne muscular dystrophy models for preclinical studies and deciphering tissue repair mechanisms
​
10:00-11:00 Student talks 5 & 6
11:00-11:30
Coffee Break
11:30-12:00
Sonia Albini, Genethon
Profibrotic iPSC-derived MYOrganoids as a translational model for gene therapy evaluation in Duchenne Muscular Dystrophy
12:00-13:00 Student talks 7 & 8​
13:00-14:00
Lunch
14:00-14:30
Sumitava Dastidar, UCL
Human Multi-Organoid Platform for Preclinical Evaluation of Neuromuscular Gene Therapies
​
14:30-15:30 Student talks 9, 10​​
15:30-16:00
Coffee Break
16:00-17:00
Gloria González-Aseguinolaza, CIMA, University of Navarra, Pamplona
Between academia and industry: juggling roles to deliver solutions to patients
​
17:00-18:30
Visit TARGET Lab
19:30-
Dinner
Friday 16 January
09:15-10:00
Yann Pereon, Rare Diseases Reference Center at Nantes University Hospital
Gene therapy treatments for patients with neuromuscular diseases​
10:00-10:45
Discussion with parent of 2 young patients
10:45-11:15
Coffee Break
11:15-12:00
Caroline Le Guiner, Target, Nantes
Gene therapy for Duchenne Muscular Dystrophy: The journey of an rAAV-µDystrophin product
​
12:00-12:15
Closing