Event will take place 10:00am - 12:00pm EST
- Dr. Bobby Gaspar, Orchard Therapeutics — Gene therapy: EMA approval, FDA status, Compassionate Use
- Dr. David Whiteman, Takeda Pharmaceuticals — Enzyme Replacement Therapy: Embolden updates
- Dr. Albert Seymour, Homology - Clinical Trial Updates
- Dr. Paul Orchard, University of Minnesota — Moderator
This final panel will allow industry leaders to interface with the MLD community and provide updates about their clinical therapies. Each individual will have 10 minutes to present an update about the status of their therapies (30 minutes of presentations). The remaining hour will be used for moderated discussion about what these updates mean for MLD patients and the community, with a focus on gene therapy. Topics covered will be Orchard’s gene therapy (EMA approval, FDA status, compassionate use),Takeda’s enzyme replacement therapy (Embolden updates), and Homology’s gene therapy development.