Cell and gene therapies (CGTs) have reached an inflection point in the speed companies are developing them and the volume coming to market. That’s according to Rachel Hatfield, PhD, managing partner at MEDiSTRAVA, who spoke recently at LSX World Congress.

GEN talked to Hatfield and colleague Dean McAlister, executive vice president at Inizio Biotech, about what this means for commercial manufacturing.

GEN: What’s the commercialization challenge for CGTs right now?

Dean McAlister
Dean McAlister

Hatfield: In its earlier stage, CGTs served a smaller population of patients and scalability was less of an issue. But now, we’re looking at [for example, treating] sickle cell disease, which affects a much larger population. So [the challenge] is ensuring that high-quality products continue to be developed and are accessible consistently to that population.

McAlister: In five to ten years, we’ll no longer be talking about whether CGTs are scalable and available for common diseases, but that depends on biotechs [staying the course] and investors staying with them.

GEN: Is the availability of CDMO slots still a challenge for companies, as it was during the pandemic?

Hatfield: There’s been a definite positive shift. [Putting] the COVID-19 issues aside, it was previously expensive and challenging to get production slots a few years ago, but now there seems to be more availability of partners, offering more cost-effective, high-quality options for companies to work with.

Rachel Hatfield, PhD
Rachel Hatfield, PhD

GEN: What factors should companies consider before using a CDMO to scale-up production?

Hatfield: I suppose you should take a long-term view about whether your organization could [build the manufacturing capacity] yourself in time, or whether you want to partner. If you’re a smaller organization driving innovation, [maybe you’re] never going to be able to fund a much larger facility yourself. So [consider] at what stage you start to talk to big pharma equivalents who are moving into gene therapy where there may be opportunities for early-stage partnerships.

GEN: What other factors affect manufacturing scale-up?

Hatfield: The practicalities and logistics of getting a substance from the manufacturing site to where it’s needed. And the reverse, in some cases. [Also], the challenge of getting the [therapy] to patients [on time], especially if you’re crossing borders or oceans.

McAlister: But, so long as the incentive for innovation is maintained, the marketplace will solve that [challenge].

Hatfield: Also, you need to understand everything from the start. Pinpoint your patient population to the point where you know what value you can bring. Then, specifically work up from preclinical stage to confirm the potential size of your market, and what your scalability [therefore] needs to be.

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