FDA Puts First Human CRISPR Study On Hold Before It Has Even Begun

(Credit: Kelly Irvine/ NIST)

U.S. regulators have put a halt on a clinical trial planned to use CRISPR technology on sickle-cell patients due to unspecified questions, CRISPR Therapeutics said on Wednesday.

CRISPR Therapeutics, who is developing the therapy, urged the FDA to green-light a study on treating sickle-cell disease in people in April.

The therapy entails extracting stem cells from a patient’s bone marrow and editing them with CRISPR in the lab. The idea is that once infused back into the patient, the edited cells would give rise to healthy red blood cells.

The FDA have ordered the company to not proceed with its study until it answers questions about its treatment — what those questions are is unknown, however. 




The FDA placed the hold “pending the resolution of certain questions that will be provided by the FDA as part of its review,” the press release said. 

The biotech uses a similar approach in a clinical trial for beta-thalassemia in Europe that’s slated to begin this year, but this therapy has not been affected by the FDA’s order. 

After the news came out, CRISPR Therapeutics stock fell as much as 19% in the after-hours on Wednesday, while the stocks of Vertex Pharmaceuticals, who are co-running the trial, remained largely unchanged, according to Business Insider. 

In other news, doctors at the University of Pennsylvania are currently enrolling patients for a (first) CRISPR trial in the U.S., where they plan on using the technology to modify human immune cells so that they become expert cancer killers.