CASEGEVY – SCI & TECH
News: US approves two gene therapies for sickle cell disease
What's in the news?
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Recently, the U.S. Food
and Drug Administration (FDA) approved a pair of gene therapies for sickle cell disease, including the first
treatment based on the breakthrough CRISPR gene editing technology.
Key details from news:
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The two gene therapies
are Casegevy and Lyfgenia.
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Both the therapies were approved for people aged 12 years and
older.
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Makers of both the
therapies have pitched them as one-time treatments, but data on how long their
effect lasts is limited.
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The only longer-term treatment for sickle cell disease is a bone marrow
transplant.
Lyf Genia:
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Bluebird bio’s sickle
cell therapy is designed to work by inserting modified genes into the body
through disabled viruses to help the patient’s red blood cells produce normal
hemoglobin.
Vertex gene therapy:
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In Vertex’s therapy,
patients must have stem cells harvested from their bone marrow.
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The cells are then sent
to manufacturing facilities where they are edited using CRISPR/Cas9 technology.
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Once the cells are
incubated, they are infused back into the patient during a month-long hospital
stay.
What is Sickle Cell Disease?
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Sickle cell disease is a
painful, inherited blood disorder that can be debilitating and lead to
premature death.
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It affects an estimated
100,000 people in the United States, most of whom are Black.
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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.
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The sickle cells tend to
stick together and can block small blood vessels, causing intense pain.
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It also can lead to
strokes and organ failure.