Upcoming Duchenne Gene Therapy Trial to Be Focus of PPMD-hosted Webinar on Wednesday, Sept. 6

Parent Project Muscular Dystrophy (PPMD) will host a one-hour webinar at 2 p.m. Eastern Time on Wednesday, Sept. 6, that will focus on an upcoming clinical trial exploring gene therapy for Duchenne muscular dystrophy.

The webinar will be led by Dr. Jerry Mendell, who, together with fellow researcher Dr. Louise Rodino-Klapac, received a $2.2 million grant from PPMD in January 2017 for their gene therapy research project at the Nationwide Children’s Hospital in Columbus, Ohio.

The project is now approaching its first human trial, expected to begin in the next few months. Mendell will talk about how the trial is designed, including inclusion and exclusion criteria for participation. He will also share planned timelines.

Those wishing to participate are asked to register and submit questions in advance. Follow this link for more information about registering and submitting questions.

The grant was the first in the nonprofit’s Gene Transfer Initiative, which intends to support research into gene-therapy-based solutions. The webinar is part of a series that intends to present researchers and companies that focus on gene therapy for Duchenne.

Such therapies include gene transfer techniques, in which a small but functional version of the dystrophin gene, referred to as micro-dystrophin, is delivered with the help of a non-infectious virus. Other approaches use gene editing with the help of the CRISPR-Cas9 system (a naturally occurring bacterial defense system that has been adapted into a gene-editing tool).

The Nationwide Children’s Hospital trial will focus on the delivery of micro-dystrophin.

But while the webinar series will present research projects in various stages of progress, it spent the first part on Aug. 15 discussing what these approaches really mean, allowing patients and families to better understand the complex science behind the therapeutic approaches. By understanding the science, PPMD hopes that families can make better choices once these therapies reach clinical trials.

PPMD also felt prompted to bring gene therapies to Duchenne patients with the recent FDA approval of Kymriah, the first gene therapy to be approved in the U.S.

While Kymriah is a cancer immunotherapy using a different approach than that likely to be used in Duchenne, the approval constitutes “another piece of evidence showing the tremendous strides this technology has made since the 1990s and its early days of research,” PPMD’s Abby Bronson wrote in a blog post.

“[The Kymriah] approval means that there are regulatory and commercial pathways for cell and gene based therapy. It means that you can put living DNA into a human and it can do its job,” Bronson wrote in her blog. “And it means that years of scientists making seemingly incremental advances can all come together and result into a giant step forward. A step forward that we believe will move this technology in a direction that will eventually benefit our community, our children.”