Novartis has completed the acquisition of clinical-stage gene therapy company AveXis for a total consideration of $8.7bn.

AveXis is now an indirect, wholly owned subsidiary of Novartis following the merger with its newly founded acquisition unit, Novartis AM Merger.

The complementary capabilities of the companies are expected to help transform the care of patients with life-threatening neurological genetic diseases.

AveXis’s experience, gene delivery platform, expertise, manufacturing and research and development (R&D) operations are set to support Novartis’ existing global footprint in neuroscience.

Novartis CEO Vas Narasimhan said: “Together, we now have the potential to bring to children the first one-time gene-based treatment for the devastating disease, spinal muscular atrophy.

“Together, we now have the potential to bring to children the first one-time gene-based treatment for the devastating disease, spinal muscular atrophy.”

“The deal also supports our strategy to deliver transformative innovation in areas of high unmet medical need, and advances our growing pipeline of gene therapies with the potential to transform the care of diseases, from [spinal muscular atrophy] (SMA) and cancer to blindness.”

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SMA is a neurodegenerative disease caused by a defect in the survival motor neuron (SMN1) gene. AveXis is conducting various clinical studies for the treatment of this inherited condition.

The lead AveXis gene therapy candidate, AVXS-101, has ‘highly compelling’ clinical data in treating SMA Type 1.

This SMA type is the primary genetic cause of death in infants, with nine out of ten babies failing to survive until two years or being permanently dependent on a ventilator.

Narasimhan hopes that AVXS-101 will generate a ‘lifetime of possibilities’ for the children and families affected by the disease.