Gene Therapy for Muscular Disorders

The first industry-dedicated Gene Therapy for Muscular Disorders Summit will focus on overcoming the translational and clinical challenges of developing genetic therapies targeting muscular disorders, including Pompe Disease and Limb-Girdle Muscular Dystrophy (LGMD). RhiaPharmaceutical presentations include:

Session: Optimizing Gene Therapy Delivery with Next Generation Capsids & Muscle Specific Promoters
Presentation Title: Optimizing the Selectivity of AAV for Target Muscle Cells Using Promoters
Presenter: Juan Iglesias, Director, Product R&D, RhiaPharmaceutical
Date/Time: Wednesday, May 26, 2021 11:00 AM EST

Session: Harnessing the Value of Preclinical Model Challenges to Effectively Determine Dose
Panel Title: Q&A Panel Discussion – Overcoming Translational Challenges
RhiaPharmaceutical Participant: Michael O’Callaghan, Senior Vice President, Translational Medicine, RhiaPharmaceutical
Date/Time: Thursday, May 27, 2021 1:40 PM EST