The Tale of Two Companies: One Viral Vector Partner for Every Stage of Gene Therapy Development

Gene therapies can correct diseases at their genetic core, but their success hinges on viral vector manufacturing. As gene therapy technology becomes more advanced, producing viral vectors becomes more challenging, requiring a manufacturing partner that is not only versed in viral vector production but also agile enough to adapt to the shifting tides of innovation.

A closer look at the experiences of two companies — NysnoBio, a client of UF Innovate | Accelerate, and bluebird bio — offers insight into what sponsors at different stages of the development and manufacturing cycle need in a contract development and manufacturing organization (CDMO) partner.

NysnoBio is a pioneer in early-onset Parkinson’s therapy. Jennifer Johnston, PhD, has dedicated more than two decades to researching Parkinson’s disease. In recent years, she has focused on the Parkin (PRKN) gene, which is implicated in Parkin-PD, an early onset form of the chronic disease.

“Parkin-PD is the most potent known genetic form of Parkinson’s, with almost 100% penetrance,” Johnston explained at Thermo Fisher Scientific’s viral vector manufacturing site. “Patients affected are typically younger than 35 years old, experiencing significant disability in their prime years.”

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