Peter Marks, who’s responsible for the FDA’s oversight of cell and gene therapies, expects regulators around the world will be better at collaborating on gene therapy approvals within the next five years, and doing so could make those treatments more accessible.
Success would look like “much more regulatory convergence globally,” Marks said Wednesday at a Reagan-Udall Foundation event. “So we actually are able to have relatively rare disease gene therapies that don’t just make it to a United States population or European Union population, but one application could potentially satisfy global regulators.”
That level of convergence could take the burden off regional approvals or even lead to a “kind of a reference approval, such as what we do for vaccines or pre-qualification with WHO,” Marks added.
Forecasting a decade into the future, he predicts a transition from AAV-based gene therapies to more CRISPR-based therapies will be “transformational because the cost should drop very significantly.”
Marks compared AAV-based manufacturing to “fine French cooking. You’ve got to have everything right.” He also noted “we’re not making the progress we’d like to see” in AAV-based manufacturing.
Whereas for CRISPR-based editing, Marks said the process “could be more like a McDonald’s approach,” in which a manufacturer still gets everything right and “the cost could go significantly down.”
Part of that cost equation includes greater adoption of synthetic biology and artificial intelligence to off-target checking, he added.
As far as the risk calculation with respect to gene therapies, Marks said that calculus can change depending on how large the patient population is, with the agency allowing more risk for smaller groups.
“Larger populations have different risk considerations, and they have to because if we unleash a gene therapy on a large number of people, and it turns out to have even a 1% or 2% chance of something very seriously adverse happening, we could set the whole field back pretty significantly,” he said. “In the smaller populations, we’ve already seen setbacks like this, and because of the benefit considerations, the number of people involved seems to be more accepted.”