The deal announced this week ups Swiss biopharma giant Novartis presence in the gene therapy space, adding clinical stage firm AveXis for approximately $8.7bn.
If it goes through, Novartis would gain breakthrough-designated candidate AVXS-101, an intended one-time gene replacement therapy for spinal muscular atrophy (SMA), which it aims to submit to the US Food and Drug Administration (FDA) for review in the second half of 2018.
According to AveXis, AVXS‑101 is made using adherent human embryonic kidney (HEK293), which have been used successfully to manufacture numerous other gene therapy candidates that have been tested or are currently being tested in other clinical trials to date.
The firm uses an adherent cell culture approach to produce AAV9 vectors it says have greater surface areas to potentially increase productivity relative to traditional flat stock approaches.
“The acquisition would also accelerate our strategy to pursue high-efficacy, first-in-class therapies and broaden our leadership in neuroscience,” said Novartis CEO Vas Narasimhan. “We would gain with the team at AveXis another gene therapy platform, in addition to our CAR-T platform for cancer, to advance a growing pipeline of gene therapies across therapeutic areas.”
With the approval of Kymriah (tisagenlecleucel) last year, Novartis is one of two firms to have a commercialised chimeric antigen receptor (CAR) T-cell therapy on the market.
And through this deal Novartis will gain access to AveXis’ gene delivery platform as well as an established manufacturing facility, located in Illinois, US.