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What they do.

AveXis is developing a gene therapy drug for Spinal Muscular Atrophy (SMA), the leading cause of genetic infant mortality. SMA is a neurodegenerative disease characterized by loss of motor neurons leading to progressive muscle atrophy and weakness.

Status:

PBM was the first investor to fund AveXis’ gene therapy program and invested prior to Phase 1 clinical trial. Today, AveXis is a publicly traded company (NASDAQ:AVXS) developing gene therapy for patients with rare and life-threatening neurological genetic diseases, including SMA.

Achievement of Note:

AveXis’ drugs have been used to treat many babies with SMA to date.

How they‘re improving the quality of peoples lives:

AveXis is developing a drug that could change the lives of babies with a truly horrific disease. The impact is manifold, from extending and improving their lives from natural history progression to giving parents hope that their children could live a longer and less challenging life.