In 2018, Sarepta Therapeutics put RNA and gene therapies at the heart of its plans for the future. The decision led Sarepta to invest in infrastructure, including gene therapy manufacturing capacity.
Working with contract manufacturing organizations (CMOs) Thermo Fisher Scientific and Paragon Bioservices, now part of Catalent, Sarepta established gene therapy capacity to support its growth plans. However, with the size of the Roche business now behind its Duchenne muscular dystrophy (DMD) gene therapy, Sarepta thinks the capacity may prove insufficient for its newly scaled up ambitions.
Sarepta CEO Douglas Ingram outlined the situation in a presentation at the J.P. Morgan Healthcare Conference.
Ingram said, “This Roche alliance will provide us with an opportunity to expand the number of patients that could be treated. And of course, the question then becomes, but you didn't build your capacity with that breadth in mind. And that's true.”
When it was developing SRP-9001 unpartnered, Sarepta was likely to focus its limited resources on key Western markets. The support of Roche positions Sarepta to more quickly target other markets.
China is an example of a market Sarepta may now target. There are around 60,000 DMD patients in China, according to Ingram, and these patients lack good treatment options.
Getting SRP-9001 to those patients could unlock a significant market opportunity but the number of gene therapy doses needed to serve China could overload Sarepta’s current capacity. Those dynamics may lead Sarepta to add new capacity.
Ingram said, “We are going to have to sit with our partner, Roche, [and] think about places around the world that we haven't even envisioned on our own to be able to get to it in a reasonable period of time. As we think that through, if that requires us to increase capacity over time, and I suspect it will, then we'll just simply do that.”
Sarepta’s confidence in its ability to add capacity is underpinned by its experience to date. Ingram sees assay and process development as “the real rate limiter” in gene therapy manufacturing, adding that “the actual ability to scale beyond that is something we can certainly do.”