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FDA Approves Second Gene Therapy for Hemophilia B

Pfizer reported a list price of $3.5 million for its new gene therapy, Beqvez, for adults with this rare bleeding disorder. ....

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US Watchdog Lifts Price Estimate for Gene Therapies

An influential US drug pricing watchdog raised the price estimate of two experimental gene therapies from Vertex Pharmaceuticals/CRISPR Therapeutics and bluebird bio for sickle cell disease. ....

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Gene Therapy for Hemophilia A 'Transforming and Liberating'

One year after infusion of a gene-transfer product, patients with the bleeding disorder had significant improvements in clotting factor production and dramatic reductions in factor concentrate use. ....

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