Matt Hoffman

PF-06939926 is a recombinant adenoassociated virus serotype 9 (AAV9) capsid carrying mini-dystrophin, a shortened version of the human dystrophin gene, under the control of a muscle-specific promoter.

Axovant reported positive safety data as well as improvements in a number of measurements of motor function and dyskinesias in patients with Parkinson disease who received treatment with their investigational gene therapy.

New early-stage data suggest that vector‐mediated gene silencing of striatal CaV1.3 expression may hold promise for preventing the induction of levodopa-induced dyskinesias in Parkinson disease.

In a pair of phase 2 trials and a phase 1 study, patients with SMA types 1 and 2 treated with the gene therapy displayed a number of motor milestone achievements and a prolonged event-free survival rate.

The microdystrophin gene transfer therapy from Solid Biosciences has shown protein expression in the first 3 patients dosed. As a result, the company is expediting the planned dose-escalating activities.

Axovant Sciences has announced that, in addition to receiving positive feedback from the FDA on its clinical development, it is expecting data from the first 2 patients dosed to read out in March 2019.

The agency is anticipating upward of 200 INDs per year by 2020 and between 10 and 20 cell and gene therapy approvals per year by 2025.

Therapies designed to treat neurologic conditions have made up 25% of the submissions to the FDA for a Regenerative Medicine Advanced Therapy designation.

The president of the ANA offered his perspective on these novel genetic therapies, as well as other a few other areas of interest.

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.