FDA clears way for faster personalized gene editing therapy

The advent of Crispr has made it theoretically possible for scientists to develop cures for many of the 7,000 rare diseases, a small fraction of which have treatments. Photo by Toondelamour/Getty Images/Postmedia files

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A top United States regulator plans to unveil a faster approach to approving custom gene-editing treatments, a move designed to unleash a wave of industry investment that will yield cures for patients with rare diseases.

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Vinay Prasad, who oversees gene therapies at the Food and Drug Administration, said scientific advances, like Crispr, have forced the agency to relax some of its strict rules. As an example, he cited the case of 10-month-old KJ Muldoon, who this year became the first person in history to have his genes custom edited to cure an inherited disease.

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“Regulation has to evolve as fast as science evolves,” Prasad said in an interview with Bloomberg News. The agency is “going to be extremely flexible and work very fast with the scientists who want to bring these therapies to kids who need it.”

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Prasad plans to publish a paper in early November outlining the FDA’s new approach. He predicted it will spark interest in developing treatments for conditions that may affect only a handful of people.

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“The moment we publish our paper, the investment in this space will flow,” Prasad said. “It will turn the spigot on.”

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The shares of companies working on gene editing rose on the news. Crispr Therapeutics AG rose as much as eight per cent, Editas Medicine Inc. climbed as much as 12 per cent and Intellia Therapeutics Inc. increased as much as 9.1 per cent

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The team behind Baby KJ’s treatment appears to be the first to benefit.

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In a paper published Friday, Rebecca Ahrens-Nicklas, a physician at the Children’s Hospital of Philadelphia, and Kiran Musunuru, an expert in genome editing at the University of Pennsylvania, explained how the new process is working for them. They’re designing clinical trials they can use to gain FDA approvals with a gene-editing platform that can be tweaked for patients with different genetic mutations.

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Historically, every treatment for every disease has needed its own separate study. The advent of Crispr, the Nobel Prize-winning gene-editing technology, has made it theoretically possible for scientists to develop cures for many of the 7,000 rare diseases, a small fraction of which have treatments. But the FDA’s demands made it seem too expensive and time-consuming.

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Now, the FDA is allowing a combined trial of patients with related genetic disorders, Ahrens-Nicklas and Musunuru wrote in the American Journal of Human Genetics. It’s a fundamental shift in how the agency approaches genetic therapies, potentially freeing them from traditional rules that slowed innovation.

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“It allows for the amazing ability to tackle multiple genetic diseases at once,” Ahrens-Nicklas said in an interview.

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The researchers said they took the unusual step of sharing their interactions with the FDA to show drugmakers there’s a regulatory path for custom-made Crispr treatments. The result may be one-time therapies that cost a few hundred thousand dollars, Musunuru said, rather than the current multimillion dollar price tags.