Story excerpt provided by Columbus Business First.
Written by Carrie Ghose.
Nationwide Children’s Hospital’s stock in one of its gene therapy spinoffs shot up to $27.4 million in value this week after the company released encouraging news from the FDA about its path toward seeking approval of a treatment for the top genetic killer of infants. The holdings are triple what they were worth in February.
AveXis Inc. reported that the U.S. Food and Drug Administration had approved a 20-patient safety and effectiveness trial for an injection that delivers a crucial missing gene in infants with the most severe form of spinal muscular atrophy, who typically don’t live past age 2. AveXis also said that when an earlier 12-patient study at the Columbus hospital is finished, the FDA has encouraged “discussion of whether the data from the Phase 1 study might provide the substantial evidence necessary to support a marketing application.”
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Originally published November 4, 2016.