RESEARCH TRIANGLE PARK – North Carolina’s global gene therapy leadership was boosted today with the announcement that AveXis, Inc., is doubling its planned workforce and investing $60 million more in its new Durham County manufacturing facility.

During a news conference at the North Carolina Biotechnology Center, state and company officials announced that the Illinois-based clinical-stage company developing gene therapies for neurological genetic diseases will create 200 additional jobs, expanding upon its May 2018 announcement that it was investing $55 million to build the new Durham manufacturing facility and creating 200 jobs there.

Avexis logo


AveXis, headquartered in Bannockburn, Illinois, was purchased in 2018 for $8.7 billion by Swiss drugmaker Novartis.

AveXis’ initial product candidate, AVXS-101, provisionally known as ZOLGENSMA (onasemnogene abeparvovec-xioi), is an investigational gene replacement therapy to treat spinal muscular atrophy (SMA) Type 1. ZOLGENSMA is designed to address the genetic root cause of SMA Type 1, a deadly neuromuscular disease with limited treatment options. ZOLGENSMA previously received Breakthrough Therapy designation and has been granted Priority Review by the FDA, with regulatory action anticipated in May 2019.

“Our primary focus is to bring gene therapies to patients suffering from devastating rare neurological genetic diseases, such as SMA, genetic amyotrophic lateral sclerosis and Rett syndrome,” said Eamon Fitzmaurice, plant controller for the Durham AveXis site.

“Continued investment in our infrastructure in North Carolina will allow us to manufacture multiple gene therapies simultaneously, helping us reach more patients, faster. Gene therapy manufacturing requires a highly skilled team, and Research Triangle Park is an ideal location for our continued expansion as it enables us to recruit top talent, including through partnership with local schools and colleges.”

The North Carolina Department of Commerce and the Economic Development Partnership of N.C. (EDPNC) led the state’s support for the company’s expansion. NCBiotech provided technical due diligence for the project, one of several recent projects that adds depth to the state’s life science cluster in the emerging area of gene therapy.

Novartis-owned AveXis bringing $55M gene therapy factory, 200 jobs to Durham

AveXis will create a variety of new positions in Durham County, adding scientists, engineers, analysts, manufacturing and operations personnel, with salaries averaging $72,952. That’s more than 6 percent higher than the current Durham County average wage of $68,731.

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 that could be worth as much as $645 million if certain milestones are met.

Samulski joined Pfizer in Sanford as vice president of gene therapy after its purchase of 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.

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 the grant, the project will grow the state’s economy by an estimated $1.3 billion. 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 $1,447,500, 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. AveXis must also remain in full compliance with its May, 2018 JDIG to receive payments from today’s grant. 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 expand 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 $483,000 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.  More information on the state’s economic tier designations is available here.

Partnering with N.C. Commerce and the EDPNC on this project were the North Carolina General Assembly, the North Carolina Community College System, the North Carolina Biotechnology Center, Durham County, and the Greater Durham Chamber of Commerce.

The brand name ZOLGENSMA has been provisionally approved by the FDA for the investigational product AVXS-101, but the product itself has not received marketing authorization or Biologics License Application approval from any regulatory authorities.

(C) N.C. Biotechnology Center