Thank you to our speakers, sponsors, and delegates who joined us in Boston for the summit!
There is newfound hope for millions with muscular diseases thanks to thanks to Sarepta's ground-breaking treatment new treatment, Elevidys, for Duchenne muscular dystrophy (DMD). This landmark achievement has sparked a wave of innovation, with numerous companies accelerating their efforts to bring similar therapies to patients with a variety of muscular diseases.
This is why we are excited to reunite 60+ industry leaders at the 4th Gene Therapy for Muscular Disorders Summit in April, the only end-to-end meeting focussing on gene therapies for muscular disorders, to tackle the most pressing challenges and together, seek solutions to realize these novel therapeutic potentials for patients in need.
What Did You Miss in 2024?
Across 3-days, we will dive into 16+ data-driven presentations and discussions surrounding:
Latest technologies in gene editing, vector, and cargo optimization
Safer, dose-effective, and targeted therapies a therapeutic reality
Immunogenicity and toxicity concerns with deep-dives into effective patient monitoring
Demonstrating efficacy through appropriate endpoint selection supported by relevant biomarkers
New data from REGENXBIO on clinical outcome selection & validation for muscular gene therapies, intended to bridge the gap between functional measures and clinical biomarkers
Recent advances in the use of MYOrganoids at Genethon as a translational model to evaluate gene therapy products for DMD
In collaboration with Regeneron, our event partner, you will be joined by trailblazers including Kate Therapeutics, Astellas Gene Therapies, Hansa Biopharma, Solid Biosciences, who face similar challenges like yourself in developing these life-changing therapies. And we will hone into their lessons learned from preclinical research to navigating human trials and approvals to help you expedite clinical development and get it first-time-right.
World-Class Speaker Faculty Included:
Morten Sogaard
President & Divison Head of Gene Therapy Research & Technical Operations
Astellas Gene Therapies
Previously Attending Companies Include:
Other Events in the Gene Therapy Series:
