Gene and cell therapies are an emerging therapy class. Here, GEN talks to Nneka Onwudiwe, PharmaD, PhD, the PRO/PE Regulatory Review Officer for the FDA, about current and future challenges for manufacturing regulation.

GEN: What do you feel are the main areas where the gene and cell therapy industry are working to meet regulatory requirements?

Onwudiwe: The manufacturing processes [in these industries] are in their infancy, so a lot of validation is needed. [This is the case,] for example, with starting materials, especially if a manufacturer is starting with a small batch and is scaling up ready for commercialization.

Even when a product makes it to clinic, there are lots of other issues that need to be considered. For example, how do you store it within a critical range of temperatures? And how do you ensure safe delivery to clinical sites?

GEN: What do you think is the biggest challenge facing manufacturers in this sector?

Onwudiwe: Capacity constraints is the biggest challenge. If regulations require certain manufacturing processes or additional testing, that becomes a bottleneck if the capacity isn’t there—as the manufacturer hasn’t anticipated it.

GEN: How would you recommend manufacturers respond?

Onwudiwe: It’s important for companies to engage with the health authority at an early stage because the last thing a manufacturer wants to find out is that either their manufacturing process is not in compliance or there’s some issue with sourcing raw materials.

The FDA has put out several guidances around cell and gene therapy, but they’re not prescriptive. And, so, manufacturers still need to engage [with the agency] because there may be nuances around their molecule or development process. As these therapies are in their infancy (20 years in this space is still new) then there are things we are still learning. Manufacturers need to have adequate resources to anticipate challenges and a designated team of regulatory professionals who really understand the regulatory pathway for their molecule and can engage with the FDA at each step.

GEN: Where do you see future challenges arising?

Onwudiwe: I think there’s a shift, not only in the biotechnology industry, but across other industries too in terms of sourcing much-needed raw materials [due to global supply chain issues]. That’s where I see the challenge in the next couple of years. And that will remain a challenge even if manufacturers have a good understanding of the manufacturing process for their molecule and it’s been validated by the FDA.

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