When Tim Hunt was invited to a meeting with Robert F. Kennedy Jr. in early March, the Department of Health and Human Services (HHS) secretary had only been sworn in a few weeks earlier.
But RFK Jr. made it clear to the room of assembled regenerative medicine industry insiders that cell and gene therapies were high on his agenda.
“He said, ‘Look, I'm in listening mode. We want to accelerate this field. We want to remain the global leaders and we're open to ideas on how to drive things forward,‘” remembered Hunt. “It was a very healthy exchange of ideas.”
As CEO of the Alliance for Regenerative Medicine (ARM)—which represents over 400 members, including biotechs—this was music to Hunt’s ears. And the good news didn’t stop at that initial meeting. In follow-up communications with HHS, it has been “consistent throughout” that cell and gene therapies are “highly aligned” with RFK Jr.’s Make America Healthy Again movement.
“They view it as getting at the root causes of diseases, as opposed to managing these oftentimes life-threatening or life-altering disorders that are also very expensive to the healthcare system,” Hunt told Fierce in an interview.
In June, Hunt was invited to participate in a gene therapy roundtable hosted by the FDA, where RFK Jr. asked the assembled scientists and advocates to provide a list of regulations they’d like to see gone.
As the leading international advocacy organization for the benefits of engineered cell therapies and genetic medicines for patients, the ARM has “constant engagement” with the FDA, according to Hunt, who spoke to Fierce on the sidelines of the Jefferies Global Healthcare in London this week.
The dialogue culminated in a meeting with FDA Commissioner Martin Makary, M.D., and Center for Biologics Evaluation and Research Director Vinay Prasad, M.D., in July. Last week, Makary and Prasad published a highly anticipated article outlining a “plausible mechanism pathway” that could offer a new way to market for personalized therapies.
The article received a “wildly enthusiastic” reception at ARM, said Hunt.
“We’ve spoken to the FDA over the past many months about the importance of what we call ‘gene editing as a platform.’ And that's in the exact vein of what they put out,” he said.
The pathway outlined in the article is expected to significantly scale the recent success of a single-patient treatment for a patient known as baby KJ into a widely available regulatory approach and offer new hope for accessible treatments among the estimated 300 million patients with rare genetic disorders.
The “broader cell and gene community” had been hopeful that the pathway could be applicable to gene therapies for conditions with thousands of patients, said Hunt, “and from what we can tell, that's the case.”
Stakeholders have already pointed out that the article is light on the specifics of how the pathway would actually work and Hunt agreed that the “next chapter” will be turning this ambition into something concrete.
“The next step is we want to translate that impressive vision into some guidance, or some very specific things that can be driven all the way down to the reviewer level [so] companies and academic centers can execute against the vision,” he told Fierce.
Despite the warm words, not every gene therapy developer has had an easy time with the FDA recently. Earlier this month, the agency made a U-turn on uniQure’s one-time gene therapy candidate for Huntington’s disease, announcing that it no longer agreed that data from a phase 1/2 study of the much-hyped candidate, dubbed AMT-130, were enough to support a regulatory application for approval.
“Everyone was surprised” by the FDA’s decision, according to Hunt. But he suggested it’s too soon to assume that AMT-130—which some analysts have described as a potential game-changer—will never make it to patients.
“This one feels to me like it's going to get another look in some way, shape or form,” he said.
The past year has seen other less encouraging news on the gene therapy front—from bluebird bio’s sale to private equity firms after the disappointing commercial performance of its three lead assets, to limitations on the use of Sarepta Therapeutics’ Duchenne muscular dystrophy therapy Elevidys following reports of liver injury and death.
Over the summer, Prasad was pushed out of the FDA amid an aggressive campaign by patient advocates, only to be quickly hired back into the same position.
When presented with some of these setbacks, Hunt—who at one point served as chief corporate affairs officer at CRISPR gene-editing company Editas Medicine—explained why he takes a long-term view.
He described how the industry’s interest in gene therapies has gone through “three recent phases,” beginning with “a period of a lot of exuberance” from 2014 to 2019 where “if you could spell ‘gene therapy’ you could make a lot of money.”
During the pandemic, cash from non-specialist investors “came flooding in” on the back of widespread enthusiasm for drug developers, leading to a situation that had “many of the hallmarks of a bubble.”
Since then, the “pendulum has swung the other way,” according to Hunt, resulting in a “kind of irrational pessimism” toward gene therapy.
It means Hunt and the ARM are now “trying to be more active and say, ‘Look, we don't need Pollyanna, and we don't need irrational pessimism.”
“We want to put it down the middle and have, you know, a disciplined but healthier growth cycle,” he added.