Sarepta Therapeutics, Aldevron to Partner on Gene Therapy Development

January 4, 2019

The partnership provides Sarepta with capacity and manufacturing slots for GMP-grade plasmid production for its micro-dystrophin Duchenne muscular dystrophy gene therapy program, as well as plasmid capacity for future gene therapy programs.

On Jan. 2, 2019, Sarepta Therapeutics, a commercial-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapeutics for rare neuromuscular diseases, and Aldevron, a producer of custom nucleic acids, proteins, and antibodies, announced that they have entered into a long-term strategic relationship to supply Sarepta with plasmid DNA needed for Sarepta’s gene therapy clinical trials and commercial supply.

Under the terms of the agreement, Aldevron will provide GMP-grade plasmid for Sarepta’s micro-dystrophin Duchenne muscular dystrophy (DMD) gene therapy program and Limb-girdle muscular dystrophy (LGMD) programs, as well as plasmid source material for future gene therapy programs, such as Charcot-Marie-Tooth, Mucopolysaccharidosis type IIIA, Pompe, and other central nervous system diseases.

“One of our highest priorities is building a robust supply chain and scalable manufacturing that can accelerate and ensure robust patient access to our pipeline of promising gene therapies on an accelerated timeline,” said Doug Ingram, president and CEO, Sarepta Therapeutics, in a company press release. “This agreement is anticipated to provide sufficient plasmid supply to support our ambitious development and commercial gene therapy objectives.”

Aldevron supplies plasmid DNA and gene editing enzymes to biopharmaceutical researchers developing gene-based medicines. The company has three locations in Fargo, ND, Madison, WI, and Freiburg, Germany. The company’s largest plasmid DNA manufacturing facility, a 70,000-ftstructure, is located in Fargo.

Source: Sarepta Therapeutics