On May 6th 2016, Dr. Chady Hakim, Ph.D., of the University of Missouri – Columbia presented exciting new data on Solid GT’s AAV micro-dystrophin program at the 19th Annual meeting of the American Society of Gene & Cell Therapy (ASGCT).
The presentation reinforces the potential of our AAV micro-dystrophin candidate to treat DMD and builds an important foundation for future clinical investigation.
Data from the new study showed that AAV micro-dystrophin gene therapy significantly improved skeletal and cardiac muscle function, reduced muscle loss and improved muscle force in preclinical models. The data also showed that a single administration of the AAV micro-dystrophin resulted in long-term dystrophin expression throughout the entire body, a very important factor in developing a successful gene therapy.
“The presentation at ASGCT marked the first time that we were able to show that our micro-dystrophin is efficacious in both small and large animal models with DMD,” said Joel Schneider, Ph.D., director of Research & Development at Solid Biosciences. “More excitingly, it represented the first time that we were able to showcase its potential to drive functional improvement in a scientific forum. The Proof of Concept established in this study serves as an extremely important foundational step for our clinical studies, which we plan to begin next year.”
The data set presented at the conference is from one of several animal studies, all of which are geared toward informing a clinical program that will yield the data necessary for regulatory review in the most expeditious and responsible way possible. These human studies will be critical to truly understanding the candidate’s efficacy and safety and its potential to improve the lives of people with DMD.
To learn more about our Research and Development efforts,