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Battling chronic ailments. Evolving science. Improving quality of life. Even helping people get a better night’s sleep. Our portfolio companies are positively impacting the world every day with their visionary people and life-changing products.
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.
PBM was the first institutional investor to fund AveXis’ gene therapy program and invested prior to commencement of the 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 life-changing drug for 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.