You may recognize the name Sarepta because of their work in gene therapy for Duchenne muscular dystrophy (DMD) and Limb-girdle muscular dystrophy (LGMD). Sarepta has entered into a long-term relationship with Aldevron. Aldevron will supply plasmid DNA to fulfill Sarepta’s needs for its gene therapy clinical trials and commercial supply. This is an important agreement as it provides Sarepta with a reliable supply of plasmid DNA.

At 70,000 square feet, Aldevron operates the largest plasmid DNA GMP manufacturing facility in the world. They were founded in 1998 and supply plasmid DNA and gene editing enzymes to biopharmaceutical researchers developing advanced gene-based medicines.

Sarepta also has programs for Charcot-Marie-Tooth, MPS IIIA, Pompe and other CNS diseases. These programs are in anywhere from discovery to clinical in their pipeline. Their Pompe program is in the discovery phase, which is like a pre- pre-clinical phase program.


Tagged: gene therapy