- Sarepta Therapeutics on Wednesday announced a long-term deal with biotech manufacturer Aldevron for supply GMP-grade plasmids, an effort to support its ambitions for gene therapy.
- Sarepta aims to bolster its supply chain and scalable manufacturing for its micro-dystrophin Duchenne muscular dystrophy and limb-girdle muscular dystrophy gene therapy programs, as well as secure supply of plasmids for future gene therapy programs.
- Aldevron makes plasmid DNA and gene editing enzymes for biopharmaceutical companies developing gene-based medicines. No financial details were disclosed.
Over the last year, Sarepta has built up its gene therapy manufacturing network around its micro-dystrophin Duchenne muscular dystrophy (DMD) candidate — a program key to the biotech's future prospects.
In June 2018, Sarepta signed a long-term strategic manufacturing partnership with Brammer Bio, and in October, the gene therapy developer inked a deal with contract development and manufacturing organization Paragon Bioservices to add extra commercial gene therapy manufacturing capacity This latest deal helps address another part of Sarepta's supply chain.
"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 Sarepta CEO Doug Ingram in a Jan. 2 statement.
Manufacturing is a key step in the successful development of gene therapies — a fact Sarepta is well aware of after the Food and Drug Administration temporarily halted its microdystrophin DMD program last year.
The hold, which was lifted within months, had been put in place after discovery of trace levels of DNA fragments in plasmids used by Nationwide Children's Hospital, Sarepta's partner, in a Phase 1/2a study of the therapy.
Plasmids are small circular DNA molecules which can be used to help transfer genes into cells for gene-based therapeutics.
Sarepta's microdystrophin program is very early, but promising results have led investors to count it as an integral part of how the biotech could address DMD.
In October, the company presented data from a small study showing the treatment helped four boys with DMD stand up, walk and climb stairs more quickly than normally would be expected.
Sarepta's only marketed product Exondys 51 (eteplirsen) won approval from the FDA in 2016, amid a massive lobbying campaign by patient groups and lawmakers. The nod sparked an uproar as the agency overruled its own expert advisory panel and staff in clearing the drug.