Tag Archives: The Stem Cell Blog

What Should Gene Therapy Cost? – PLoS Blogs (blog)

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2017-10-26 NEWS

PLoS Blogs (blog)

What Should Gene Therapy Cost?
PLoS Blogs (blog)
By the time that the FDA’s advisory committee gave a near-instantaneous and unanimous thumbs-up for gene therapy to treat a form of hereditary blindness on October 12, tears were freely flowing. Fittingly, it was World Sight Day. The new drug, Luxturna

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2017-10-26 NEWS

PharmaTimes

Shire’s haemophilia A gene therapy wins Orphan status
PharmaTimes
Shire’s gene therapy candidate SHP654, an investigational factor VIII gene therapy for the treatment of haemophilia A, has been awarded Orphan Drug Designation by US regulators. The move qualifies Shire for access to various incentives designed to …