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Lambert J and Marks DP. Featured fireside chat: FDA perspective. Presented at: Cell & Gene Meeting on the Mesa (virtual); Oct. 12-16, 2020.

October 29, 2020
3 min read

COVID-19, manufacturing challenges limit cell and gene therapy progress, FDA official says


Lambert J and Marks DP. Featured fireside chat: FDA perspective. Presented at: Cell & Gene Meeting on the Mesa (virtual); Oct. 12-16, 2020.

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Progress in the field of gene and cell therapy continues with rapid development despite the negative impact of COVID-19 on research into novel treatments, according to the director of FDA’s Center for Biologics Evaluation and Research.

Peter Marks, MD, PhD, delivered this assessment during the virtual Cell & Gene Meeting on the Mesa.

CAR T-cell reserarch at the NIH.
Source: H. Darr Beiser.

“This is a very challenging time,” he said.

Marks estimated that a year ago, he spent up to 75% of his time on gene and cell therapies. Now, he said he spends about 80% of each day on COVID-related issues — including vaccines, convalescent plasma and polyclonal antibodies.

Marks still reserves some time for cell and gene therapy, “but not anywhere near as much time as I would want,” he said.

Peter Marks, MD, PhD
Peter Marks

“What keeps me up at night is worrying about what happens when we see the light of day and COVID clears,” Marks said. “I’m worried about the phase 3 clinical trials or pivotal trials in gene therapies that are not going optimally. I worry about the delays we have had in moving ahead some of the advances in manufacturing.”

His office continues to process investigational new drug applications and meet with new applicants, but Marks admits the pandemic has slowed the pace of development and presents his office with a challenge.

“I’m hoping that as this winter approaches and we start to see vaccines approved [for COVID-19], that we will be able to get back to focusing more on gene and cell therapies,” he said.

Beyond the COVID effect

Several cell or gene therapies have been approved by the FDA, and many others are in various stages of development. Nevertheless, the pandemic has altered the trajectory of new therapeutics for rare diseases.

“It’s clear that COVID-19 has adversely affected all aspects of the development of cell and gene therapies,” Marks said.

The extent of the virus’s impact varies based on the product, he added.

“It’s also clear that manufacturing remains a rate-limiting factor in the production of cell and gene therapies,” Marks said.

The primary manufacturing challenge is the lack of a reproducible process scale-up from clinical trial-sized lots to commercial-sized lots.

“Because the manufacturing of these therapies has become such a challenge, the process itself has become very proprietary,” Marks said. “There is much less dynamic exchange of information, which is preventing the field from advancing as quickly as it could.”

The solution, according to Marks, is “quality manufacturing of advanced therapies from the outset.”

This especially true for gene therapies that achieve dramatic outcomes, such as onasemnogene abeparvovec-xioi (Zolgensma, Novartis Gene Therapies) for spinal muscular atrophy — one of the five FDA-approved gene or cell therapies.

Marks said the manufacturing process must be in place from the start. Otherwise, time is lost having to translate it from the research lab to the commercial setting.

“It’s lost time, and lost opportunities for patients to benefit,” he said.

‘Individualized medicine’

The gene and cell therapy industry will need to streamline its processes to achieve high-quality, advanced manufacturing capabilities, Marks said. Currently, the industry exists in a “sweet spot” that allows it to deliver gene therapies to smaller populations.

“If we are to start delivering gene and cell therapies to larger populations, it will require quantum improvements in vector and manufacturing technologies,” Marks said. “There are literally hundreds of diseases that could be addressed with gene therapy now if we could figure out a way to sustainably make those therapies in terms of cost and production. A number of these diseases are devastating and occur in developing children.”

Marks said one of his office’s goals is to effectively address these types of diseases.

“If we can do this for many small populations over and over again, then we will start to learn a lot more about critical quality attributes and key manufacturing parameters. That exercise will benefit the entire gene and cell therapy field and will eventually yield benefits for a larger group of patients,” Marks said.

“I think we are moving from personalized medicine, where you find the right drug on the shelf to treat the patient, to individualized medicine, where we create the right drug to treat the patient,” he said.