US approves two gene therapies for sickle cell disease (updated)

Crispr Therapeutics AG

08/12/2023 16:21

(Adds details in paragraphs 3-6)
       Dec 8 (Reuters) - The U.S. Food and Drug Administration
on Friday approved two gene therapies for sickle cell disease
from Vertex Pharmaceuticals  VRTX.O  and CRISPR Therapeutics
 CRSP.BN , as well as from bluebird bio  BLUE.O .
    Sickle cell disease is a painful, inherited blood disorder
that can be debilitating and lead to premature death. It affects
an estimated 100,000 people in the United States, most of whom
are Black.
    In sickle cell disease, the body makes flawed, sickle-shaped
hemoglobin, impairing the ability of red blood cells to properly
carry oxygen to the body's tissues. 
    The sickle cells tend to stick together and can block small
blood vessels, causing intense pain. It also can lead to strokes
and organ failure.
    Vertex's treatment, to be sold under the brand name Casgevy,
is approved for patients ages 12 and older with the most severe
form of the disease. The company has said it has the potential
to be a one-time treatment.
    Bluebird's product will be sold under the brand name
Lyfgenia and is also approved to treat patients 12 years of age
and older. 
    
  
    

 (Reporting by Sriparna Roy, Leroy Leo and Bhanvi Satija in
Bengaluru;
Editing by Caroline Humer and Bill Berkrot)
 ((Sriparna.Roy@thomsonreuters.com;))