FROM 1440 NEWS
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Sickle Cell Gene Therapy
The US Food and Drug Administration yesterday approved a pair of gene therapy treatments for sickle cell disease, a group of inherited red blood cell disorders caused by mutations in a gene that produces an oxygen-carrying protein known as hemoglobin. One of the treatments, called Casgevy, is based on breakthrough CRISPR technology—a Nobel Prize-winning gene-editing tool (see 101, w/video)—and is the first approved gene-editing therapy in the US.
Codeveloped by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy involves harvesting cells from a patient’s bone marrow, editing the cells in a lab using CRISPR technology, and inserting the modified cells back into the patient’s body. Read one man’s account of being among the first people to experience the CRISPR-based treatment here.
The second treatment, developed by Bluebird Bio and called Lyfgenia, uses a harmless virus to introduce a gene into a patient’s body to help produce a hemoglobin substitute. Both Casgevy and Lyfgenia have been approved for people 12 years and older.
Sickle cell disease (see overview) affects more than 100,000 Americans per year and 20 million people worldwide.
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OPINION
I received the new gene-editing drug for sickle cell disease. It changed my life.
- As a patient enrolled in a clinical trial for Vertex’s new exa-cel treatment, I was among the first to experience CRISPR’s transformative effects.
By Jimi Olaghere
December 4, 2023
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The options for treating sickle cell disease are very limited. Denying access to such a powerful and transformative treatment based on someone’s ability to pay, or where they happen to live, strikes me as unethical. I believe patients and health-care providers everywhere deserve to know that the treatment will be available to those who need it.
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Truly it was an arduous journey.
And, while it may be hard to understand, breakthroughs are expensive. But as soon as they are made, the inventors and others work on making it available cheaply and easily. Indoor plumbing, electric light, and automobiles quickly became available to the masses thanks to the “invisible hand” of the marketplace and the incentives af making a profit.
It would be better IMHO for him to focus on the Gooferment and its incestuous relationship with Big Pharma as the barrier to quicker progress.
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Reinke Faces Life 