Novartis announces takeover of AveXis

Novartis has announced the acquisition of AveXis, a company specialising in gene therapies for rare and orphan neurological genetic diseases.

AveXis is currently studying a number of potential treatments for spinal muscular atrophy (SMA), a serious inherited neurodegenerative disease. The lead AveXis gene therapy candidate, AVXS-101, has shown significant potential as a treatment for SMA type 1, the number one genetic cause of death in infants.

AVXS-101 has already received breakthrough therapy designation in the US, EU and Japan, with an expected US launch date in 2019. If approved, it would be a first-in-class single-use therapy that fixes the root cause of SMA by effectively replacing the defective gene responsible.

Vas Narasimhan, chief executive officer of Novartis, said: "We believe AVXS-101 could create a lifetime of possibilities for the children and families impacted by this devastating condition."

AveXis also offers advanced AAV9 gene therapy manufacturing capabilities and research facilities, including investigational therapies for Rett syndrome and a genetic form of amyotrophic lateral sclerosis.

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