As of June 2021, the FDA has received more than 900 gene therapy applications for investigations – but only two heritable diseases currently have FDA-approved gene therapies.
Gene therapies hold tremendous promise for patients, and technologies like CRISPR-Cas9 are poised to create a new wave of medicines – but how can we ensure they actually get to patients?
Luca Issi, Sr Biotech Research Analyst at RBC Capital Markets Wealth Management, moderated a discussion with experts including:
- Will Chou, CEO of Aruvant Sciences, Inc.
- Lawrence Klein, PhD, COO of CRISPR Therapeutics AG
- Barbara Lavery, Chief Program Officer of Alliance for Cancer Gene Therapy
- Guarav Shah, CEO of Rocket Pharma
Where are we in the innovation cycle and where are we going next?
“Overall, it’s still a young market,” said Aruvant Sciences’ Chou. “There are going to be more wins, and when those happen, there’s going to be a different psychology in the market.”
One challenge is human talent: “This is a people business, an expertise business,” he continued. “There will be more positions than experienced people, but with more time, people will get experience.”
“We’re really excited about the potential for gene editing to introduce a step change for what these gene therapies are able to achieve clinically,” said CRISPR Therapeutics’ Lawrence Klein. “CRISPR gene editing can usher in a new wave of therapeutics.”
Both a challenge and opportunity is the “sheer volume of new modalities, new approaches, and things coming out of academia that have great promise," said Alliance for Cancer Gene Therapy’s Barbara Lavery. "But how do you track those into the commercial setting and take them all the way to patients?”
There’s an “enormous amount of creativity and innovation coming down the pike,” but we have a lot of work to do to get to product approval," she added.
What about safety?
“Safety’s certainly on everyone’s mind,” especially with regards to some sickle cell therapies, said Chou.
“A really fantastic job has been done with the transparency of these investigations.”
“The number of patients treated with CRISPR-based therapies is in the hundreds,” with “zero side effects minor or major attributable to the gene editing process,” said Klein.
“There can be a tendency to think of the genome as this immutable, never-changing object,” but “no two cells in your body have the exact same genomic material,” he continued.
A sunburn would lead to “more DNA damage than we’ll ever produce with treating a patient with one of these CRISPR-based therapies that we’ve developed with the stringent require we adhere to.”
How should we think about durability and sustained efficacy?
The Alliance for Cancer Gene Therapy’s Lavery brought up the newly released 10-year data on the first CAR T patients treated, which showed that cells are still present 10 years later.
“In solid tumors, the biggest thing we’re lookin at is the tumor micro-environment,” she said. We’re “just at the beginning of the work in solid tumors.”
So, what’s next?
“In the next 10-12 years we’ll actually be able to apply it to more common, chronic conditions,” predicted Klein.
“For the first time in the history of our species, we’re talking about not therapies but cures and I think people don’t quite see that point yet,” said Rocket Pharma’s Guarav Shah.
Once, people didn’t believe this was possible, but “we are so far past that now, we’re going to see so many more new successes,” said Chou. Expect a “sea change.”