Tough road ahead for Bluebird Bio despite FDA approval for sickle cell therapy

The Food and Drug Administration’s approval on Friday of two gene therapies for sickle cell disease sets up what could be an intense competition between Bluebird Bio, maker of Lyfgenia, and the partnership of Vertex Pharmaceuticals and CRISPR Therapeutics, whose drug is Casgevy.

For the first time, physicians and patients will have a choice of potentially curative treatments for an inherited disease that affects nearly 100,000 Americans.

The Vertex/CRISPR team would seem to have the edge. As the first CRISPR-based therapy, Casgevy has the advantage of being powered by the latest and buzziest biological technology, though some physicians and patients may be more comfortable with the lentiviral vector technology behind Lyfgenia, which dates back to the 1990s — even with a “black box” warning of a risk of blood cancer added to its label.

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

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

$30

for 3 months, then $39/month

$30 for 3 months Get Started

Then $39/month

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

Get unlimited access to award-winning journalism and exclusive events.

Subscribe

Log In