Contribute Try STAT+ Today

A one-time hemophilia A treatment from Pfizer (PFE) and Sangamo Therapeutics (SGMO) showed promising results in a small, early-stage study, pointing to a possible future in which patients might have a handful of curative options for the rare bleeding disorder.

In an ongoing 10-patient trial, the treatment boosted the production of factor VIII, a protein that is key to forming blood clots and is lacking in people with hemophilia A, according to data presented Saturday. Three patients getting the highest dose — the one Pfizer and Sangamo plan to use in future trials — saw their factor VIII levels returned to normal, and bleeds were reduced to zero. A fourth patient was dosed too recently to draw any conclusions.

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

GET STARTED

What is it?

STAT+ 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
  • STAT+ Conversations
  • Weekly opportunities to engage with our reporters and leading industry experts in live video conversations
  • 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.

Your daily dose of news in health and medicine

Privacy Policy