Welcome to the 4th Gene Therapy for Muscular Disorders Summit
Delivering safe and effective muscular gene therapies to patients
June 2023 saw the approval of Sarepta’s DMD gene therapy, the first ever to treat a muscular disorder. This landmark approval has ignited a wave on innovation in the field with several companies hot on their heels to progress their treatments into and through the clinic for a range of muscular indications, with a sharp focus on safety and efficacy.
The 4th Gene Therapy for Muscular Disorders is returning to the community once again in April 2024, covering the most pressing challenges across preclinical development, translation into the clinic, and navigating the first-in-human trials and pivotal studies to develop transformative gene therapies for a range of muscular diseases for which there are little or no alternative treatment options. With a recent approval in the space, there is no better time to unite the community to help progress treatments into the clinic and through approvals for a range of muscular indications with a sharp focus on safety and efficacy.
World-Class Speaker Faculty Includes:
Senior Director - Clinical Outcomes Research
Vice President - Pharmaceutical Development
Global Franchise Lead Gene Therapy