The first clinical trial of CRISPR-Cas9 sponsored by U.S. companies has launched, testing the genome-editing technique in patients with the blood disorder beta thalassemia, according to an announcement posted Friday on the U.S. clinical trials website.

The Phase 1/2 clinical trial, co-sponsored by Vertex (VRTX) Pharmaceuticals and using an experimental treatment from CRISPR Therapeutics, will be conducted at a single hospital in Regensburg, Germany, and aims to recruit up to 12 adults with the inherited disease. Although it was only a matter of time before the start of the first company-sponsored CRISPR clinical trial, Editas Medicine’s experimental treatment for a rare form of blindness was widely expected to be the first in the clinic.

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

GET STARTED

What is it?

STAT Plus is STAT's premium subscription service for in-depth biotech, pharma, policy, and life science coverage and analysis. Our award-winning team covers news on Wall Street, policy developments in Washington, early science breakthroughs and clinical trial results, and health care disruption in Silicon Valley and beyond.

What's included?

  • Daily reporting and analysis
  • The most comprehensive industry coverage from a powerhouse team of reporters
  • Subscriber-only newsletters
  • Daily newsletters to brief you on the most important industry news of the day
  • Online intelligence briefings
  • Frequent opportunities to engage with veteran beat reporters and industry experts
  • Exclusive industry events
  • Premium access to subscriber-only networking events around the country
  • The best reporters in the industry
  • The most trusted and well-connected newsroom in the health care industry
  • And much more
  • Exclusive interviews with industry leaders, profiles, and premium tools, like our CRISPR Trackr.

Leave a Comment

Please enter your name.
Please enter a comment.

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

Privacy Policy