Gene therapy for Duchenne is centered on the goal of successfully introducing a smaller, but efficient version of dystrophin into the muscle cell. INS1201 is an investigational gene therapy product, utilizing an adeno-associated virus (AAV9) to transport a shortened version of the dystrophin gene (micro-dystrophin) that may provide benefit in place of the missing full-length dystrophin protein. INS1201 is delivered through an Intrathecal Injection.
A Phase 1 study is actively recruiting.
This program is sponsored by Insmed Gene Therapy.
For information regarding current or upcoming clinical trials please visit our Explore Clinical Trials Page.
