- Sarepta Therapeutics has signed up to a long-term strategic partnership with Paragon Bioservices, a move aimed at further strengthening its gene therapy manufacturing network.
- The deal will add additional commercial manufacturing capacity for Sarepta's micro-dystrophin Duchenne muscular dystrophy (DMD) gene therapy, but also provides a platform for future gene therapy programs, Sarepta said. No financial details were released.
- Paragon has two facilities in Maryland. The second, which is under construction, is a 151,000 square-foot, GMP gene therapy biomanufacturing facility located in Anne Arundel County. It is expected to come online in February 2019.
Manufacturing can be a real stumbling block for gene therapy companies, which face numerous challenges to successfully produce the DNA-altering medicines.
Some biotechs have chosen to build their own manufacturing facilities, betting on the increased control that comes with owning production. Others have mixed in contract manufacturers. Both approaches, though, can run into issues.
Sarepta Therapeutics knows this well, having seen a Phase 1/2a trial of its microdystrophin gene therapy program put on clinical hold earlier this year. The Food and Drug Administration requested the halt after an analysis found trace levels of DNA fragments in research-grade plasmids supplied by an undisclosed third party to Nationwide Children's Hospital, which runs the study./p>
While the clinical hold was lifted quickly, it's a clear example of both the importance of reliable manufacturing and the caution of the FDA as gene therapy programs advance.
Sarepta's deal with Paragon Bioservices will give the biotech access to commercially-scalable capacity for its micro-dystrophin program, which has shown promising initial signs of efficacy in four boys with DMD.
A contract development and manufacturing organization, Paragon Bioservices is experienced in gene therapy manufacturing, and recently opened a new gene therapy-focused facility. The partnership will also include Sarepta's future programs, which also include Limb-girdle muscular dystrophy, glycogen storage disease type II and disorders of the central nervous system.
Earlier this year, Sarepta inked a similar manufacturing deal with Brammer Bio for commercial supply for its micro-dystrophin gene therapy product.
For Doug Ingram, Sarepta's president and CEO, the deals are a means to accelerate development of its pipeline and to complement the company's own internal efforts.
Correction: A previous version of this article incorrectly stated Paragon Bioservices' plant under construction would be its third located in Maryland.