AveXis wins the inside track at the FDA with gene therapy ‘breakthrough’
Chicago-based AveXis has lined up the latest breakthrough therapy designation from the FDA, gaining an inside track at the agency for its early-stage gene therapy for lethal cases of spinal muscular atrophy in children. The biotech says it won that mark of distinction after regulators got a preliminary look at the results from their Phase I study.
AveXis was able to score a $95 million IPO last February as it joined the first wave of biotechs to hit the public market this year, a little after the once red-hot gene therapy field had begun to cool somewhat. The offering followed a $65 million crossover round last fall which was joined by Roche.
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