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 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: 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.”

Having pioneered the basic biochemistry and therapeutic potential for the Parkin gene, Johnston founded NysnoBio in 2019 to take aim at this target. With a goal of developing a Parkin gene replacement therapy, NysnoBio is in the early stages of its journey and their challenges are manifold: navigating the gene therapy landscape, ensuring their work's reproducibility, and scaling production without compromising quality.

Their efforts got a significant boost in July of 2023, when the company was awarded a research grant from the Michael J. Fox Foundation for Parkinson’s Research.

Like many emerging companies, NysnoBio doesn’t have the employees, infrastructure, or manufacturing space to develop and manufacture their therapy. Therefore, the team opted to work with a CDMO to support the work. “For us, partnering with a CDMO that has a strong track record in viral vector development and process engineering makes the most sense,” Johnston explained.

Their search led them to Thermo Fisher Scientific. “Our challenge is ensuring that our work is reproducible and scalable, so we need a collaborator familiar with the nuances of early-stage gene therapy, one who can evaluate our project from every angle and offer guidance and reliability,” Johnston explained. “We also need a partner not just for now, but for the future, which is why we chose Thermo Fisher.”

bluebird bio: Scaling up and reaching out

With an established portfolio focusing on severe genetic diseases, bluebird bio already has treatments that have made significant impacts on conditions like β-thalassemia and cerebral adrenoleukodystrophy (CALD). In December 2023, the company received FDA approval for its one-time gene therapy for sickle cell disease.

Given their focus on the later phases of development, bluebird bio’s partnership needs revolve around scaling their operations, ensuring consistent quality, and optimizing supply chains for a broader patient base.

The supply chain consideration is essential, according to Tom Klima, the company’s Chief Commercial and Operating Officer. “When you think of gene therapy and the supply chain, you're taking a piece of a patient and making it part of your supply chain. It's a huge mistake to think that you can only start thinking about this later in development. All the decisions you make early in development have a huge impact as you get closer to commercialization.”

Furthermore, the responsibility toward patients is paramount. Klima explains, “Often, if there are mistakes along the way, people forget that there's a doctor and a patient at the other end. You need to call and explain why their cells were not produced properly. Having a trusted partner like Thermo Fisher, who understands that they're a massively important part of patient care, is super important.”

As therapies progress and target larger patient populations, the scale of manufacturing has to evolve concurrently. “With CALD, we started with a smaller population of about 40 patients, but then moved to a bigger population with β-thalassemia of about 1,500 patients,” Klima said. “Now, we're looking at sickle cell disease, where there are about 20,000 patients that could benefit from gene therapy.”

For an established company like bluebird bio, the partnership with a CDMO is less about guidance and more about competence, Klima stressed. “We need a partner to handle large-scale productions, ensuring the quality remains uncompromised as quantities increase.”

Finding the right partner for your project

To navigate the complexities of gene therapy development, partnering with an experienced CDMO is crucial for both emerging and established companies. Whether tackling early-stage challenges or scaling up for commercialization, the “right” partner can provide essential support and innovation when and how you need it.

Learn more about how Thermo Fisher Scientific can support your gene therapy journey.