AveXis, Inc.

The European Medicines Agency was this week deciding whether the planned EU marketing authorization applications (MAAs) for three investigational products deserved accelerated assessments – apitegroma

Novartis was a pioneer in gene therapy with the successful development of Zolgensma (onasemnogene abeparvovec-xioi) for spinal muscular atrophy. And while some big pharmas have backed away from the sp

Long periods of follow-up may be necessary for gene therapies, particularly if and when the US Food and Drug Administration is willing to grant a company an accelerated approval for such a treatment,

Long periods of follow-up may be necessary for gene therapies, particularly if and when the US Food and Drug Administration is willing to grant a company an accelerated approval for such a treatment,