Sophie Olivier

Company: Lysogene

Job title: CSO

Seminars:

Phase 2-3 gene therapy trial using adeno-associated virus vector for patients with mucopolysaccharidosis type IIIA 10:50 am

• A gene therapy approach for MPSIIIA is promising when delivered directly to the brain enabling transduction of neurons, which in turn can lead to sufficient levels of active enzyme to cross-correct neighboring cells. • AAVance is a Phase 2-3, single-arm, international study of AAV serotype rh.10 carrying the human SGSH (AAVrh.10-SGSH) for the treatment…Read more

day: Day Two