Two patients with a rare inherited disease are showing early signs of improvement after being injected with a groundbreaking genome-editing fix from Sangamo Therapeutics.

That’s the encouraging news emphasized by the biotech on Wednesday from its latest clinical trial, the first in which gene-editing is being used in patients to try to repair disease-causing DNA. But Sangamo is simultaneously downplaying other results from the study that are more confounding and may give outsiders reason to be concerned.

Unlock this article by subscribing to STAT Plus and enjoy your first 30 days free!


What is it?

STAT Plus is a premium subscription that delivers daily market-moving biopharma coverage and in-depth science reporting from a team with decades of industry experience.

What's included?

  • Authoritative biopharma coverage and analysis, interviews with industry pioneers, policy analysis, and first looks at cutting edge laboratories and early stage research
  • Subscriber-only networking events and panel discussions across the country
  • Monthly subscriber-only live chats with our reporters and experts in the field
  • Discounted tickets to industry events and early-bird access to industry reports

Leave a Comment

Please enter your name.
Please enter a comment.

  • To which person (and company to whom this person is affiliated) has the Court of Appeals “awarded” the Foundational Crown Patent for the “Gene Editing Process, cas 9” ? To Ms. Doudna of Intellia, Inc., Ms. Charpentier of Crisper Therapeutics AG, or Mr. Zhang of Editas Medicine, Inc. and on what date ?

Sign up for our Daily Recap newsletter

A roundup of STAT’s top stories of the day in science and medicine

Privacy Policy