An investigational hemophilia A treatment from Spark Therapeutics reduced bleeds by nearly 100 percent in a small clinical trial, improving upon earlier data that left investors unsatisfied. But it still remains to be seen whether Spark’s gene therapy can beat out a rival effort underway at BioMarin.

In a study on 12 patients, Spark’s one-time treatment boosted the production of factor VIII, a protein that is key to forming blood clots that is missing in people with hemophilia A, according to data released Tuesday. The highest dose — the one Spark is going to use in late-stage trials — increased average factor VIII levels to 30 percent of normal, the company said, keeping patients entirely free of bleeding in the process.

Unlock this article by subscribing to STAT Plus. Try it FREE for 30 days!

GET STARTED

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.

Sign up for our Daily Recap newsletter

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

Privacy Policy