Skip to Content
Biotechnology and health

FDA halts one of the first human CRISPR studies before it begins

A trial planning to use the gene-editing tool CRISPR on sickle-cell patients has been put on hold because of unspecified questions from US regulators.

Background: CRISPR Therapeutics, which is developing the therapy, sought approval from the US Food and Drug Administration in April to begin the study. The therapy involves extracting stem cells from a patient’s bone marrow and editing them with CRISPR in the lab. The idea is that the edited cells, once infused back into the patient, would give rise to healthy red blood cells.

FDA hold: But according to a statement on Wednesday from CRISPR Therapeutics, the FDA ordered the company not to proceed with its study until it answers questions about its treatment (the company didn’t provide any further details about what those questions are, however).

The FDA did not immediately respond to a request for comment.

Planned trials: CRISPR Therapeutics says a European trial for the inherited blood disorder beta thalassemia, which involves a similar procedure, won’t be affected by the FDA’s order. The company plans to begin that study in the second half of the year.

Separately, the first CRISPR trial in the US, conducted by the University of Pennsylvania, is currently enrolling patients.

Deep Dive

Biotechnology and health

How scientists traced a mysterious covid case back to six toilets

When wastewater surveillance turns into a hunt for a single infected individual, the ethics get tricky.

An AI-driven “factory of drugs” claims to have hit a big milestone

Insilico is part of a wave of companies betting on AI as the "next amazing revolution" in biology

The quest to legitimize longevity medicine

Longevity clinics offer a mix of services that largely cater to the wealthy. Now there’s a push to establish their work as a credible medical field.

There is a new most expensive drug in the world. Price tag: $4.25 million

But will the latest gene therapy suffer the curse of the costliest drug?

Stay connected

Illustration by Rose Wong

Get the latest updates from
MIT Technology Review

Discover special offers, top stories, upcoming events, and more.

Thank you for submitting your email!

Explore more newsletters

It looks like something went wrong.

We’re having trouble saving your preferences. Try refreshing this page and updating them one more time. If you continue to get this message, reach out to us at customer-service@technologyreview.com with a list of newsletters you’d like to receive.