Pfizer latest to test gene therapy for Duchenne muscular dystrophy

Pfizer (PFE) has begun its first human trials of a gene therapy to treat Duchenne muscular dystrophy, following two other companies that have launched trials and one treatment for the disease in recent years.

The first patient received the experimental therapy, called PF-06939926, on March 22, Pfizer said Thursday. The study is expected to run through 2019 and to enroll about a dozen boys from 5 to 12 years old at four sites.

The participants will receive an infusion containing a virus that will deliver a shortened version of the dystrophin gene. That replacement gene will produce a protein that keeps muscle cells intact.

STAT+ Exclusive Story

Already have an account? Log in

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

Already have an account? Log in

Individual plans

Group plans

Monthly

$39

Totals $468 per year

$39/month Get Started

Totals $468 per year

Starter

$20

for 3 months, then $399/year

$20 for 3 months Get Started

Then $399/year

Annual

$399

Save 15%

$399/year Get Started

Save 15%

11+ Users

Custom

Savings start at 25%!

Request A Quote Request A Quote

Savings start at 25%!

2-10 Users

$300

Annually per user

$300/year Get Started

$300 Annually per user

View All Plans

To read the rest of this story subscribe to STAT+.

Subscribe

Log In