Effectively Navigating High Gene Therapy Doses and Optimize Systemic Deliver to Transduce Target Muscle Cells and Create Meaningful Genetic Therapies with Strong Safety & Efficacy Profiles

"Intimate setting and size made it possible to meet with everyone and have open and constructive conversations"

Sharon McGonigle, Vice President – R&D, Solid Biosciences

"A very informative meeting with good opportunity to network with leaders in the gene therapy for muscular disorders space"

Abhinav Adhikari, Scientist, Epic Bio

"High quality presentations with lots of cutting-edge technology"

Greg Luerman, Vice President – Research & Partnerships, Curi Bio

"I had a unique opportunity to learn about the highs and lows of current gene therapies, which will help me to provide our patients with the information that will allow them to develop realistic expectations regarding treatments and using these therapies. The in-site and pre-meeting customer staff interactions were excellent."

Dorota Gruber, Assistant Chief – Paediatric Cardi-genomics, Cohen Children’s Medical Center


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:

4th Gene Therapy for Muscular Disorders 2024 FAVICON

Latest technologies in gene editing, vector, and cargo optimization

4th Gene Therapy for Muscular Disorders 2024 FAVICON

Safer, dose-effective, and targeted therapies a therapeutic reality

4th Gene Therapy for Muscular Disorders 2024 FAVICON

Immunogenicity and toxicity concerns with deep-dives into effective patient monitoring

4th Gene Therapy for Muscular Disorders 2024 FAVICON

Demonstrating efficacy through appropriate endpoint selection supported by relevant biomarkers

4th Gene Therapy for Muscular Disorders 2024 FAVICON

New data from REGENXBIO on clinical outcome selection & validation for muscular gene therapies, intended to bridge the gap between functional measures and clinical biomarkers

4th Gene Therapy for Muscular Disorders 2024 FAVICON

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:

Previously Attending Companies Include:

sarepta logo
solid biosciences logo
nationwide childrens hospital logo

Other Events in the Gene Therapy Series: