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Sarepta eyes a different approach to Duchenne: Gene Therapy

Posted on: 10 January 2017, source: Business Journal
After winning approval in September for the first-ever Duchenne muscular dystrophy drug, Cambridge-based Sarepta Therapeutics is broadening its approach to treating the disease through a pair of gene therapy programs. Sarepta (Nasdaq: SRPT) announced Tuesday that it had reached an agreement with Ohio-based Nationwide Children’s Hospital to exclusively license a pre-clinical program that seeks to treat DMD through gene therapy. The company also said it had entered into a research agreement with the hospital to develop a second pre-clinical gene therapy program, with an exclusive option to license it later.


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Sarepta’s Exondys 51, the first-ever DMD treatment to be approved in the U.S., targets a specific Duchenne muscular dystrophy gene mutation shared by about 13 percent of patients. The two gene therapy programs unveiled Tuesday have the potential to help all patients suffering from the disease, the company said. Both are expected to enter clinical tests in 2017.

Exondys 51 is a so-called exon-skipping drug, meaning it is designed to “skip over” a gene mutation that prevents the creation of dystrophin, a protein needed for muscle maintenance. Gene therapy involves treating diseases by replacing a faulty gene with a corrected one. Some other biotechs are already taking a gene therapy approach to DMD, including Cambridge-based Solid Biosciences and Bamboo Therapeutics, a North Carolina-based firm that was acquired by Pfizer (NYSE: PFE) in August.

The gene therapy program that Sarepta is exclusively licensing, called Galgt2, involves inducing genes that make proteins that can mimic dystrophin. The second program is backed by a $2.2 million grant from Parent Project Muscular Dystrophy, an advocacy and research group. The announcement came shortly before Sarepta CEO Ed Kaye was scheduled to give a presentation at the J.P. Morgan Healthcare Conference, an annual gathering of biotech industry leaders and investors in San Francisco.

Sarepta is one of several Duchenne-focused biotechs in the Cambridge area, a group that also includes Akashi Therapeutics, Catabasis Pharmaceuticals (Nasdaq: CATB) and Summit Therapeutics (Nasdaq: SMMT). Akashi said recently that it anticipates having three drugs to treat DMD in clinical trials this year, all of which are designed to help patients preserve muscle function regardless of their genetic makeup. That suggests that the drugs, if successful, could be used in conjunction with Exondys 51. Summit and Catabasis also have the goal of treating all patients with DMD.

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Posted on: 10 January 2017, source: Business Journal
After winning approval in September for the first-ever Duchenne muscular dystrophy drug, Cambridge-based Sarepta Therapeutics is broadening its approach to treating the disease through a pair of gene therapy programs. Sarepta (Nasdaq: SRPT) announced Tuesday that it had reached an agreement with Ohio-based Nationwide Children’s Hospital to exclusively license a pre-clinical program that seeks to treat DMD through gene therapy. The company also said it had entered into a research agreement with the hospital to develop a second pre-clinical gene therapy program, with an exclusive option to license it later.
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