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Posted 05 November 2020 | By Michael Mezher 

Marks fields questions on cell and gene therapy development

2707 Expect new guidance from the US Food and Drug Administration (FDA) on the manufacture of CAR-T cells, said Peter Marks, director of the agency’s Center for Biologics Evaluation and Research (CBER).
Marks answered questions on a range of issues related to cell and gene therapy development during a webinar hosted by Genetic Engineering & Biotechnology News.
Saying that CBER is working on new guidelines for CAR T-cell therapy manufacturing, Marks said, “The agency is trying to put out some more clear information on what we’re expecting for CAR T-cells. I’m not sure exactly when it’s going to appear, but stay tuned,” Marks said.
On the topic of how FDA would regulate cell and gene therapies manufactured at the point of care, Marks explained that it would be possible to regulate the device that makes the therapy, rather than requiring a biologics license application (BLA) for each location that has the device.
“There the issue is the device will allow you to make a product at the point of care and the clearance or the approval of that device … would take care of that,” Marks said. “On the other hand, the more complicated issue, for instance, this concept of whether you could have CAR T-cells made at the point of care. There it’s a much more challenging situation.”
Marks explained that two potential models would be licensing the individual sites or having a primary sponsor that holds the license and the point-of-care facilities become “subsites for manufacturing” under the license.
Either way, Marks said, “This is one of those areas where if you’re thinking about going down this path it’s probably good to come into the agency for a high-level discussion about the key issues that might go into it.”
According to Marks, the most common question related to cell and gene therapy manufacturing the agency gets is how companies can go from one generation manufacturing process to the next through different stages of product development.
“We are very open to having process evolution, but the issue…is sometimes people with their first generation process get really great clinical data and then they’re in a situation where they have to move to a new process to scale-up, and that scale-up creates a problem because they lose activity, they can’t bridge when they need to do a bridging trial … and they’ve made a major process change,” Marks said.
Marks’ advice to manufacturers thinking about those issues is to, “Think ahead, to the extent that you can, with your process development and try to move to your process that you want to use for your commercial product as quickly as possible.” Otherwise, Marks encouraged cell and gene therapy developers to have a reasonably validated plan to move to larger scale commercial processes.
On the topic of potency assays, Marks stressed the importance of using a validated assay before Phase 3 trials. “We really prefer if you can have a validated potency assay when you’re in Phase 3. It’s really, really helpful on the back end,” he said. “We occasionally have people who get to the end who can’t get their potency assay validated and that ends up becoming an issue.”
For those looking to incorporate new technologies into their development programs, such as next-generation sequencing and high throughput systems for viral control, Marks encouraged companies to request a CBER Advanced Technology Team meeting. “We’re very open to having meetings on those platforms,” Marks said, adding that such meetings are “incredibly useful.”
“Because they’re informal, we find that sponsors tend to gain a lot and we actually tend to gain a lot because we learn about people’s new technologies,” he said.
Genetic Engineering & Biotechnology News


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