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Sickle cell cure will be cost-effective if health disparities considered -study finds

By Nancy Lapid

(Reuters) - Multimillion-dollar gene therapies being tested for sickle cell disease will be cost-effective in the United States if health disparities tied in part to decreased access to healthcare are taken into account, researchers said at a meeting of blood-disease specialists.

In the United States, about one of every 365 Black babies is born with sickle cell disease - a genetic disorder in which misshapen blood cells cause strokes, organ damage, severe pain and early death - according to the U.S. Centers for Disease Control and Prevention (CDC).

Roughly 100,000 Americans have the disorder, the CDC estimates, with millions more affected worldwide.

After decades in which the disorder was largely ignored by major drugmakers amid calls from patient advocates to do more, more than half a dozen companies are developing cutting edge gene therapies for sickle cell disease.

These include Novartis AG, CRISPR Therapeutics AG with Vertex Pharmaceuticals, and Sangamo Therapeutics.

Furthest along is Bluebird bio, which is expected to apply for U.S. approval of its LentiGlobin gene therapy early in 2023.

Gene therapy treatments, which are intended to be one-time cures, are likely to cost more than $2.7 million per patient, Dr. George Goshua of the Yale School of Medicine told doctors on Sunday at a meeting of the American Society of Hematology (ASH).

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