The agreement between Sarepta and Aldevron, a plasmid DNA and gene editing enzymes supplier, will provide good manufacturing practice (GMP) grade plasmid for Sarepta’s micro-dystrophin Duchenne muscular dystrophy (DMD) gene therapy program and limb-girdle muscular dystrophy (LGMD) programs.
Micro-dystrophin is a small portion of the gene, dystrophin, which individuals with DMD lack. Previous trials have shown positive results for the use of micro-dystrophin for the treatment of DMD.
Previously, Sarepta’s micro-dystrophin trial for the treatment of DMD was put on hold by the US Food and Drug Administration, after a DNA fragment was found within a plasmid used for the gene therapy. The plasmids supplied at the time were from an undisclosed third-party manufacturer.
The agreement with Aldevron will also provide plasmid source material for the company’s future gene therapy programs. Aldevron will help establish a supply chain for scalable manufacturing for Sarepta. An established supply chain, according to Doug Ingram, CEO of Sarepta, is one of the company’s strategic objectives.
Ingram further stated that, through this secure supply, “[the company] can accelerate and ensure robust patient access to our pipeline of promising gene therapies on an accelerated timeline.”