AveXis Presents Promising Clinical Trial Results of Gene Therapy for Type 1 SMA

The pharmaceutical company AveXis recently presented the latest data about its clinical trial for AVXS-101, a gene therapy for infants with type 1 SMA. The one-time infusion is designed to correct the faulty gene responsible for spinal muscular atrophy, using a harmless virus able to cross the brain-blood barrier.

MORE: Gene therapy may help babies with type 1 SMA

AveXis presented its very encouraging results at the annual meeting of the American Academy of Neurology (AAN), a six-day event in Boston.

Among the data reported, the company found that the earlier a child begins AVXS-101 treatment, the earlier the infant reaches developmental milestones. According to an SMA News Today report, a company researcher shared a video of treated babies engaged in activities largely unknown among those with type 1 disease: rolling over and crawling with ease, and sitting up and walking independently, including one who was holding a toy. That toddler was also able to reach up and touch an elevator button. Find out more about the presentation here.

AveXis met with the U.S. Food and Drug Administration (FDA) on Monday, May 1, to discuss the next steps for the therapy. According to our report, if all went well at that meeting, the company will begin a new and larger clinical trial of the gene therapy in babies with type 1 SMA in both the U.S. and Europe this year.

MORE: Genentech is considering the design of phase 3 clinical trial for SMA therapy.

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