Landmark Gene-Editing Study Advances After China Crispr Clamor

(Bloomberg) -- After a global controversy over a Chinese scientist’s surprise claims that he’d edited the DNA of human embryos, U.S. regulators blessed a small company’s quest to use the same genetic technology in a new medicine.

Editas Medicine Inc. said on Friday that the U.S. Food and Drug Administration had accepted its application to begin the first in-human study of an experimental therapy for a rare eye disorder that uses Crispr, the same technology the Chinese researcher claims to have used to alter the genetic makeup of a pair of twin infant girls before they were born.

The trial by Editas would be the first time that a Crispr-based medicine has been studied inside of living patients in an official clinical trial anywhere in the world, the company said in a statement. A number of other biotechnology companies have therapies in development that use Crispr to treat a range of diseases.

Crispr can splice away parts of the human genetic blueprint that make people susceptible to disease or defects. Scientists have generally taken a cautious approach to using it in humans given that the long-term effects of altering a person’s genes aren’t well understood. When Shenzhen-based researcher He Jiankui said this week that he’d used the tool to try to make the girls resistant to HIV, it caused an international scientific backlash.

Crispr has a host of applications, from genetically modifying crops to changing how basic biomedical research is conducted. The technology has captured the imagination of investors. Editas and rivals such as Intellia Therapeutics Inc. and Crispr Therapeutics AG have seen their shares climb despite their current lack of marketable drugs, and even though some experts believe that the therapies that use Crispr for humans are at least five years away.

Editas shares, which have risen by 90 percent since their February 2016 initial public offering, were up 0.8 percent at $30.35 at 9:37 a.m. in New York. The Cambridge, Massachusetts-based company said that with the FDA acceptance, it had earned a $25 million milestone payment from partner Allergan Plc.

The trio of companies leading the Crispr race in the U.S. are using distinct approaches for gene editing. In contrast to Editas, where genetic editing takes place “in vivo,” or inside the body, Crispr Therapeutics is primarily focused on an approach using cells that have been extracted from the body. Intellia is also working on in vivo gene editing and has yet to file for a trial approval.

Crispr Therapeutics and partner Vertex Pharmaceuticals Inc. have started enrolling patients for the first company-sponsored human trial in the U.S. in patients with severe sickle cell disease. Crispr Therapeutics also recently enrolled the first patient in a separate study of a genetic therapy for beta-thalassemia patients in Europe.

--With assistance from Tatiana Darie.

To contact the reporter on this story: Rebecca Spalding in Boston at rspalding@bloomberg.net

To contact the editors responsible for this story: Drew Armstrong at darmstrong17@bloomberg.net, Timothy Annett

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