The Food and Drug Administration on Tuesday took another step toward expanding access to treatments for rare diseases, announcing a new proposal for personalized gene therapies.
In the latest draft guidance, regulators focused on treatments designed to correct different mutations in the same disease-causing gene. Often, the individual mutations are so rare that traditional clinical trials are impractical or even impossible.
Under the plan, a drugmaker could win approval for a specific personalized gene therapy targeting one mutation using more of a traditional approach, then later win clearance for different mutations with laboratory data instead of a whole new clinical study. The parameters for possible later approvals would be laid out in what the FDA is calling an “individualized feature space” in the original application.
The promise of personalized gene-editing therapies burst into the national conversation in May 2025, when a group of scientists worked together to develop a medicine for a critically ill baby named KJ Muldoon in just a few months. But most people with rare diseases face the stark fact that developing individual treatments requiring clinical studies just isn’t profitable for drugmakers.
With the latest plan, the FDA is hoping to change the economic calculus, allowing what TD Cowen analyst Joseph Thome called a “plug and play” optionality. If enacted, this draft guidance could be especially relevant for products under development by gene editing specialist Prime Medicine, Thome wrote in a note to clients. Prime shares jumped 15% early Wednesday.
Beam Therapeutics CEO John Evans says the idea would help his company too. “All of this is completely supported by the science of base editing,” Evans wrote in a series of posts on the social media platform X, referring to Beam’s core drug development technology. “But it’s still exhilarating to see the FDA crafting such a forward-looking approach.” Beam’s shares rose 8%. READ MORE
Source: biopharmadive.com
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