Nationwide Children’s stake in gene therapy spinoff soars to $27M on positive FDA news

Gene therapist at work

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.”

Click here to read the complete article.

Originally published November 4, 2016.

Leave a Reply

This site uses Akismet to reduce spam. Learn how your comment data is processed.

%d bloggers like this: