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Sarepta Enters the Gene Therapy Business

James Radke
Published Online: Tuesday, Jan 10, 2017
Just prior to their presentation at JP Morgan Healthcare Conference, Sarepta Therapeutics announced it is partnering with Nationwide Children’s Hospital to develop 2 gene therapies to treat Duchenne muscular dystrophy.
 
The company, heavily involved in the use of exon skipping drug development to treat Duchenne muscular dystrophy, is partnering with 2 labs to develop clinical trials to test:
  1. microdystrophin gene therapy
  2. Galgt2 gene therapy. 

Both programs will be for patients with Duchenne muscular dystrophy.
 
The microdystrophin gene therapy program will be led by Jerry Mendell, MD and Louise Rodino-Klapac, PhD and the Galgt2 gene therapy program will be led by Paul Martin, PhD.

Microdystrophin Gene Therapy

The initial phase 1/2a trial should begin in late 2017 at Nationwide Children’s. Key to the development of the program was a research grant from Parent Project Muscular Dystrophy (PPMD) has $2.2 million. Sarepta has also committed to the trial for an undisclosed amount and has an exclusive option to license the program.
 
“We are thrilled Sarepta has entered into this research agreement with Nationwide. The additional resources will bolster Nationwide’s ability to conduct an even more rigorous and robust trial,” said Pat Furlong, founding president and chief executive officer of Parent Project Muscular Dystrophy.

Galgt2 Gene Therapy

This program is a “surrogate gene therapy approach” in which the gene therapy is used to make proteins that can perform a similar function as dystrophin, with the goal of producing a muscle cell that can function normally even when dystrophin is absent.
 
This approach, if successful, could be used on a number of muscular dystrophies.
 
Dr. Kevin Flanigan, the Principal Investigator leading the clinical trial said, “Our goal is to have this program in the clinic during 2017 and begin to evaluate a therapy that has the potential to treat patients of all ages and disease severity.”
 
In a Nationwide Children’s Hospital podcast, Dr Martin talked about the use of Galgt2 gene therapy. In the podcast, Dr Martin noted that the proteins made from Galgt2 gene therapy, while not dystrophin, can compensate for dystrophin’s loss by keeping the muscles functioning properly. 


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