Vertex/CRISPR one-dose gene editing therapy for sicklecell disease would be cost effective if priced at up to $1.9 million, an influential U.S. drug pricing group said on Wednesday. TwitteRx
April 13, 2023
They expect to potentially cure SCD through a single dose of the therapy, exa-cel. SCD is a genetic disorder that causes strokes, organ damage, severe pain and early death and affects roughly 100,000 people in the United States. Gene therapies have been pricey and their list prices have crossed the million-dollar mark in recent years.
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