AveXis Brings $55M Gene Therapy Factory, 200 Jobs to Durham


AveXis logo

AveXis, an Illinois-based clinical-stage company developing gene therapies for neurological genetic diseases, is investing $55 million in a new Durham manufacturing facility that will create 200 jobs. 

It’s the latest in a series of such high-tech biomanufacturing facilities being opened in North Carolina, drawn by the state’s highly trained biomanufacturing workforce and its widely respected ecosystem for the specialized sector known as precision health. 

Officials said the 200 employees at the new facility will earn an average salary of $104,000. 

AveXis was recently purchased for $8.7 billion by Swiss drugmaker Novartis. Novartis has a history of pharmaceutical manufacturing in North Carolina. In 2006 the company’s generic drug unit, Sandoz, took over operation of a manufacturing facility built in 1997 by Novopharm in Wilson. Sandoz is actively making generic pharmaceuticals there today.

More recently, Novartis sold its animal health headquarters in Greensboro to Eli Lilly in 2014, where it remains in production under the Elanco banner.

Novartis began production in 2014 from a $1 billion-plus biomanufacturing facility in Holly Springs to make a next-generation flu vaccine using a high-tech cell culture process rather than the decades-old reliance on chicken eggs for growing the key vaccine ingredient. 

DNA strand illustration
-- Shutterstock

As part of a global business realignment, Novartis sold that plant and its vaccines business in 2015 to the vaccines division of CSL Ltd., an Australian biopharmaceutical company, for $275 million. CSL renamed the vaccines business Seqirus

AveXis intends to use its new Durham facility to manufacture its first product candidate, AVXS-101, its proprietary gene therapy to treat three types of spinal muscular atrophy, or SMA. SMA Type 1 is the leading genetic cause of infant mortality.

The U.S. Food and Drug Administration, has granted AVXS-101 orphan drug and breakthrough therapy designations for all types of SMA. The FDA also provided fast track designation for the SMA Type 1 indication. 

Besides AVXS-101, AveXis also plans to develop other novel treatments for rare neurological diseases, including Rett syndrome and a genetic form of amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 (SOD1) gene.

“The transformation of gene-based technologies into economic impact in North Carolina continues to be a focus of the Biotechnology Center, not only for human health but also for animal health and agriculture,” noted Bill Bullock, senior vice president of statewide operations & economic development with the North Carolina Biotechnology Center.

“We are pleased to welcome AveXis to the growing list of world-class gene and cell therapy companies developing and producing potentially transformative products in North Carolina,” added Doug Edgeton, NCBiotech president and CEO.

NC a gene therapy leader with AveXis, AskBio, Pfizer, bluebird, G1, Locus, Precision

Other companies announcing gene therapy facilities in North Carolina recently include Pfizer, investing $100 million and adding gene therapy capabilities to its campus in Sanford, and bluebird bio, in Durham. 

North Carolina’s home-grown precision health cluster is also fueled by G1 Therapeutics, Locus Biosciences, and Precision Biosciences. Each uses a different method to attack infections, disease and/or cancer at its source – the genetic code of an organism. 

Each of those companies has received significant funding in the last year, including G1’s $105 million IPO, Locus’ $19 million funding round and Precision’s $1.6 billion deal with Baxalta. 

NCBiotech also provided early funding support to G1 and Locus.

AveXis purchased rights to its gene therapy technology in 2015 for an undisclosed sum from Asklepios BioPharmaceutical, a Chapel Hill gene therapy platform company commonly called AskBio. 

AskBio said it received an upfront payment and stands to gain milestone payments and royalties based on AveXis’ successful development and commercialization of its treatment. AveXis said it would use the technology in its Phase 1 gene transfer clinical trial in spinal muscular atrophy.

AskBio’s gene therapy platform is based on the work of Jude Samulski, Ph.D., a pioneering scientist in gene therapy who was recruited to the University of North Carolina School of Medicine from the University of Pittsburgh in 1993 with the help of about $250,000 in grant funding from the North Carolina Biotechnology Center. Samulski directed UNC’s Gene Therapy Center for many years.

Several grants and loans from the Biotech Center have supported the development of Samulski’s academic research and commercial technologies respectively.

Samulski and Sheila Mikhail, C.P.A., MBA, JD, founded AskBio in 2003 to commercialize AAV gene therapies. AskBio subsequently spun out four gene therapy companies: 

  • Actus Therapeutics, to develop gene therapies for rare genetic diseases including Pompe disease and epilepsy.
  • NanoCor Therapeutics, developing treatments for cardiovascular disease.
  • Chatham Therapeutics, developing treatments for hemophilia. Chatham was sold to Baxter International (now Shire) in 2014 for $70 million.
  • Bamboo Therapeutics, developing treatments for rare neuromuscular diseases. Bamboo was sold to Pfizer in 2016 for $150 million in a deal worth as much as $645 million if certain milestones are met. 

Samulski joined Pfizer in Sanford, N.C., as vice president of gene therapy after it purchased Bamboo Therapeutics but is transitioning back to UNC and to AskBio as the company’s scientific founder and chief science officer, Mikhail said.

Following Pfizer’s acquisition of Bamboo, the drug giant worked with NCBiotech to develop a transformative $4 million postdoctoral fellowship program in gene therapy.

The North Carolina Department of Commerce led the recruitment of AveXis to the state, in partnership with the NC Economic Development Partnership. Life science professionals at NCBiotech also provided technical due diligence and other support for the project.  

Decision enhances NC's expertise in gene therapy

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

Andrew Knudten, AveXis senior vice president of technical operations and chief technical officer, spoke for the company. 

“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,” he said. “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.” 

Commerce Secretary Anthony M. Copeland added, “It’s notable that AveXis has chosen to expand its manufacturing operations in North Carolina. Life science companies understand the many advantages our state offers manufacturers, particularly the investments North Carolina has made in education and workforce training for the biotechnology industry.”

AveXis will create a variety of positions in Durham County, including engineers, manufacturing and quality control personnel, and supply chain specialists. When all new positions are filled, the total payroll impact is anticipated to be in excess of $20 million annually.

AveXis’ expansion in Durham County will be facilitated, in part, by a Job Development Investment Grant (JDIG) approved by the state’s Economic Investment Committee earlier today. Over the course of the 12-year term of this grant, the project will grow the state’s economy by an estimated $918.5 million. 

Using a formula that takes into account the new tax revenues generated by the new jobs, the JDIG agreement authorizes the potential reimbursement to the company of up to $2,232,000, spread over 12 years. State payments only occur following performance verification by the departments of Commerce and Revenue that the company has met its incremental job creation and investment targets. JDIG projects result in positive net tax revenue to the state treasury, even after taking into consideration the grant’s reimbursement payments to a given company.

Because AveXis chose to locate in Durham County, classified by the state’s economic tier system as Tier 3, the company’s JDIG agreement also calls for moving as much as $744,000 into the state’s Industrial Development Fund – Utility Account. The Utility Account helps rural communities finance necessary infrastructure upgrades to attract future business. Even when new jobs are created in a Tier 3 county such as Durham, the new tax revenue generated through JDIG grants helps more economically challenged communities elsewhere in the state. 

Also partnering on the AveXis project were the North Carolina General Assembly, the North Carolina Community College System, Durham Technical Community College, Durham County, and the Greater Durham Chamber of Commerce.

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