October 14, 2022 -- The cell and gene therapy (CGT) sector will continue to face investment and regulator challenges in the near term. That’s the consensus of companies in the space who participated in a workshop at this week’s Cell & Gene Meeting on the Mesa (MOTM) in Carlsbad, CA.
Looking at what's next for the CGT industry in 2023 and beyond, the companies called out familiar hurdles to commercialization -- including the current investment environment -- and what they can do to mitigate growing market pressures.
One of the biggest challenges for the industry is "investment headwinds" that have continued throughout 2022, following two successive record years of financing flowing into the sector, according to a recent report from the Alliance for Regenerative Medicine (ARM).
"Meeting and exceeding regulators' expectations -- that's where we need to be to achieve these [clinical] wins," Andrew Knudten, chief operating officer at Jaguar Gene Therapy, told the MOTM audience. "That's where the industry needs to go. We need to get more wins and then the money will follow."
Stewart Abbot, PhD, chief scientific officer for Turnstone Biologics, similarly noted that a colleague once sagely said that "good clinical data solves all" in such investment challenges. Abbot contends that CGT companies are largely valued by investors on clinical data, not preclinical.
"At the end of the day, what we're really pushing toward is to get as many clinical studies done as efficiently as possible with the best design possible, because you can take a good drug with poor design and not get a good outcome," Abbot said. "Getting that clinical data efficiently and effectively is our key goal."
Robert Peters, PhD, chief scientific officer at Ensoma, called the current CGT investment environment "a moment in time" and that the industry has seen recent clinical successes with notable sector approvals from regulators for curative therapies.
"Ultimately, that will turn things around and drive more interest back into the industry," Peters said.
However, regulatory bottlenecks have also been challenging for companies as the sector looks to move toward commercialization. Abbot noted that the CGT industry has "somewhat been a victim of its own success" providing regulators with a lot more data from clinical trials with "a lot of information" flowing into the regulatory agencies.
"They're assimilating that [data] and they are now in a position to ask quite relevant questions of sponsors," said Abbot. "The bar does appear to be going higher but it's really the knowledge level is going higher."
Knudten said the expectations of the regulators "have always been there" however in prior years companies would go to regulatory agencies "asking is this good enough" and "that's not the case anymore." The bar "was always there but now you're really being held to it," he added.