Innovative therapies, such as mRNA, gene therapy and CAR-T cell therapy, have enormous potential for the treatment of children with rare diseases. Development, early phase clinical trials, regulation and the accessibility of these therapies call for cooperation and a new approach. During this symposium, we will highlight the developments inside and outside academic hospitals and we want to talk to all the experts involved about the challenges and opportunities.
This event is in Dutch.
Programma
12.45 – 13.15 Aanmelden & lunch
13.30 – 13.35 Welkom en opening door dagvoorzitter Sander Schimmelpenninck
13.35 – 14.00 Key note – Daniel de Boer
14.00 – 14.15 Internationale markt en toekomst: noodzaak tot samenwerking -Ward van Beers
Ontwikkelen: academische en industriële research en ontwerp
14.15 – 14.27 Prime Editing – Sabine Fuchs
14.27 – 14.40 ICAT – Trudy Straetemans
14.40 – 15.10 Paneldiscussie met o.a. Trudy Straetemans, Ray Schiffelers, Jeffrey Beekman en Roger Adan
Testen: uitvoering vroege fase trials in de kliniek
15.40 – 15.53 Stamcelbehandeling bij stroke – van bench to bedside – Cora Nijboer
15.53 – 16.05 030-Lab: Unieke cohorten & faciliteiten – Kors van der Ent en Sabine Michel
16.05 – 16.35 Paneldiscussie met o.a. Olaf Savelkouls, Tim Weglewski, Peter Gisberts en Michel Zwaan
Benutten: markttoelating & betaalbaarheid
16.35 – 16.47 HSCT, CAR-T – Caroline Lindemans
16.47 – 17.00 Gentherapie SMA – Ludo van der Pol
17.00 – 17.30 Paneldiscussie met o.a. Carla Hollak, Lonneke Timmers, Ton de Boer, Renske ten Ham en Lourens Bloem
17.30 Afsluiting door Sander Schimmelpenninck en borrel
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