Novartis invests $55m in US gene therapy site, prepares BLA

By Flora Southey

- Last updated on GMT

AveXis – a Novartis company – has added a manufacturing facility in North Carolina to its site network, where it will make investigational therapies for neurological genetic diseases.

The clinical-stage gene therapy firm will invest $55m into the site – located in Research Triangle Park, North Carolina, US – and add 200 jobs in engineering, manufacturing and quality control, and supply chain roles.

“As a long-established biotechnology hub that attracts the nation’s top talent, Research Triangle Park was an optimal location to expand our footprint and complement our existing state-of-the-art manufacturing site in the Chicagoland area,” ​said chief technical officer Andrew Knudten said in a statement​.

Other pharmaceutical firms to occupy sites in Research Triangle Park include GlaxoSmithKline​ and Biogen​.

BLA preparations

When Novartis acquired AveXis for $8.7bn in May this year​, it picked up the biotech’s investigational gene therapy AVXS-101, designed to treat spinal muscular atrophy (SMA).

An AveXis spokesperson told us the firm is preparing to submit the candidate to the US Food and Drug Administration (FDA) for review: “A pre-BLA [biologics license application] is scheduled with the US FDA in June surrounding the regulatory pathway for AVXS-101 for the treatment of SMA.”

“There are preclinical assets in development for the treatment of genetic ALS [amyotrophic lateral sclerosis] and Rett syndrome, for which AveXis anticipates a submission of INDs [investigational new drug] late 2018 or Q1 2019,” ​she added.

Novartis is not new to the gene therapy space. The Big Pharma company received FDA approval for chimeric antigen receptor (CAR) T-cell therapy Kymriah (tisagenlecleucel) in August last year​.

And last month​, the FDA approved Kymriah for a second indication – to treat adult patients with relapsed or refractory (r/r) large B-cell lymphoma – making it the only CAR T-cell therapy to receive US regulatory approval for two distinct indications

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