AveXis, Maker of SMA Therapy AVXS-101, Expanding Manufacturing Operations to North Carolina

AveXis is expanding its operations to North Carolina, opening up a new manufacturing center in Durham County, North Carolina Gov. Ray Cooper recently announced in a press release.

The company is anticipated to invest about $55 million into the new facility and open up about 200 new jobs, giving a boost to the state’s economy while improving its capabilities to manufacture therapies targeting rare neurological and genetic diseases, including spinal muscular atrophy (SMA).

“We’re proud to be the place where cutting edge work will happen on gene therapies to fight disease and improve people’s health,” Cooper said in the release. “North Carolina has long been a leader in the life sciences, and AveXis’ decision further enhances the state’s expertise in this emerging field.”

Illinois-based AveXis, a Novartis company, is developing AVXS-101, a proprietary gene therapy that could prove to be a transformative treatment for babies and children with SMA types 1-3.

AVXS-101 is designed to deliver a functional copy of the SMN1 gene to motor neuron cells in SMA patients. The therapy is being developed in close collaboration with the U.S. Food and Drug Administration.

Based on data from a completed Phase 1 clinical trial (NCT02122952) in SMA type 1 patients, the agency granted AVXS-101 orphan drug status for all types of SMA. It also gave the treatment breakthrough therapy status and fast track status for SMA type 1. Those designations are aimed at accelerating the therapy’s regulatory approval for the condition.

The company is currently conducting several trials, including SPR1NT Phase 3 (NCT03505099) in presymptomatic infants with SMA types 1, 2, or 3; STR1VE Phase 3 (NCT03306277) in children with SMA type 1 younger than 6 months; STRONG Phase 1 (NCT03381729) in children up to 60 months old with SMA type 2; and the European STR1VE-EU Phase 3 trial (NCT03461289) in SMA type 1 children.

All of these ongoing trials are currently enrolling patients with the exception of STR1VE-EU, which is not yet recruiting. More information is available by clicking on the trial numbers above.

AveXis is also planning the REACH trial for patients with SMA types 1-3, ages 6 months to 18 years, who are ineligible for the other studies.

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“Our primary focus is to bring gene therapies to patients suffering from devastating rare neurological diseases — such as SMA, genetic amyotrophic lateral sclerosis, and Rett syndrome — and continued investment in establishing our manufacturing infrastructure is a critical component to accomplishing this goal,” said Andrew Knudten, senior vice president of technical operations and chief technical officer of AveXis. “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.”

AveXis’ expansion, which is supported in part by a Job Development Investment Grant from the state, will create several job positions in Durham County, including engineers, manufacturing and quality control personnel, and supply chain specialists.

“North Carolina’s ongoing commitment to the biotech and life science industry continues to pay dividends,” said state Sen. Mike Woodard. “Companies like AveXis know they will find the support they need at the state, regional, and local level when they choose to do business in North Carolina.”

The North Carolina Department of Commerce and the Economic Development Partnership of North Carolina led the state’s support for the company’s expansion.