Sarepta Therapeutics enters manufacturing partnership for DMD treatment

By Maggie Lynch

- Last updated on GMT

(Image: Getty/Rost D-9)
(Image: Getty/Rost D-9)

Related tags Duchenne muscular dystrophy Manufacturing Clinical trial Gene therapy Gene therapy candidate

Sarepta Therapeutics signed a long-term, strategic manufacturing partnership with Paragon Bioservices to produce micro-dystrophin to grow its gene therapy program.

The partnership will give Sarepta access to additional commercial manufacturing capacities, which it will use for the production of micro-dystrophin, part of its Duchenne muscular dystrophy (DMD) gene therapy program.

The company may use the additional capacity for its future gene therapy programs, such as its limb-girdle muscular dystrophy​ program.

DMD is caused by a lack of the dystrophin protein that keeps muscle cells intact. With the treatment of micro-dystrophin, patients receive a small amount of the gene, dystrophin, they lack. A previous trial saw positive results​ in using micro-dystrophin as a treatment for DMD.

The company stated in a press release that it is actively working towards making its gene therapy programs stronger and will establish a Gene Therapy Center of Excellence ​in Columbus, Ohio. It also revealed that it is focused on creating partnerships, like that with Paragon, with the aim of ensuring it has a scalable manufacturing approach to accelerate its gene therapies.

Paragon employs over 300 people at two locations in Maryland and is currently constructing a 151,000-square-foot good manufacturing practice (GMP) facility for gene therapy and biomanufacturing.

Sarepta said that Paragon is consistent with the hybrid manufacturing strategy that it follows, and therefore will be a good fit to increase its manufacturing capabilities – its hybrid manufacturing strategy involves adeno-associated virus-based manufacturing​ vectors.

Recently, a trial testing Sarepta’s micro-dystrophin treatment was recently put on hold by the US Food and Drug Administration​, after a DNA fragment was found within a plasmid used in the gene therapy. The plasmids in which the DNA fragment was found were produced by a third-party manufacturer. 

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