Matt Hoffman

Since the inception of the idea more than 3 decades ago and its initial development 20 years later, Sarepta Therapeutics’ micro-dystrophin gene therapy has now made its way to human trials.

The gene therapy is also being assessed for long-term safety data, as well as an additional delivery method.

In a small trial, the AAVrh74.MHCK7.micro-dystrophin therapy has shown high levels of transduced micro-dystrophin expression.

With the planned submission of an IND for the first AAV gene therapy for the rare genetic condition, some experts are wondering if things are moving too quickly.

Across 3 cohorts, the therapy showed improvements in on-time without troublesome dyskinesia, ranging from 2.1 hours to 3.5 hours.

Renova Therapeutics is planning a 536-patient phase 3 trial, FLOURISH, to begin in early 2018.

Jeffrey Heier talks about RGX-314 gene therapy and the advantages of subretinal delivery.