NC’s Gene Therapy Leadership Based On 25+ Years of Focused Support
North Carolina is a global leader in gene therapy thanks to its renowned life science workforce development programs and the scientific output of its research universities.
For example, Pfizer’s first human trial use of its PF-06939926 gene therapy was at Duke University in 2018. The one-time intravenous gene therapy infusion is designed to treat a degenerative disorder shared by about one of every 3,500 boys at birth: Duchenne Muscular Dystrophy (DMD). Other test sites for the trial were UCLA and the University of Utah.
And this product candidate, in clinical trials conducted by the world’s largest pharmaceutical company, is the result of seminal discoveries made by Jude Samulski, Ph.D., just 12 miles down the road from Duke, at the University of North Carolina at Chapel Hill.
Samulski pioneered the use of the live, but harmless, recombinant adeno-associated virus (AAV) as the premier delivery mechanism, or vector, for gene therapy. He came to the UNC School of Medicine from the University of Pittsburgh in 1993. He’s director of UNC’s Gene Therapy Center and co-founder of the prolific RTP company Asklepios BioPharmaceutical, commonly known as AskBio.
Samulski’s recruitment was bolstered by about $250,000 in grant funding and other support to UNC from the North Carolina Biotechnology Center. All told, eight grants and loans from the taxpayer-funded Biotech Center totaling about $1.5 million have supported Samulski’s academic research and commercial technologies.
The DMD therapy was under development by an AskBio spinout, Bamboo Therapeutics of Chapel Hill, when Pfizer purchased Bamboo in 2016. The deal was for $150 million up front, with milestone payments that could be worth as much as $645 million.
Pfizer soon built a $100 million gene therapy manufacturing facility adjacent to its vaccine manufacturing plant on its 230-acre campus in Sanford. And now the global pharmaceutical giant is attaching another 100,000-square-foot, $500 million gene therapy production facility to the newly opened first phase. Read more about Pfizer's gene therapy expansion announcement.
The U.S. Food and Drug Administration approved another AskBio-derived gene replacement therapy in May. That was for AveXis’ Zolgensma, to treat spinal muscular atrophy (SMA). That’s an inherited and deadly neuromuscular disease that, like DMD, otherwise offered its victims limited treatment options.
After AveXis bought rights to Zolgensma, Novartis bought AveXis for $8.7 billion to get control of the therapy. Now AveXis, headquartered some 30 miles north of Chicago, in Bannockburn, Illinois, is investing $115 million and hiring 400 people at its new Durham gene therapy manufacturing facility.
Other gene therapy companies in the Triangle
Besides AskBio, AveXis, Bamboo and Pfizer, other gene therapy companies that have set up shop in the Triangle (and the year of their founding) include:
- Adrenas Therapeutics Inc. (2018), a California-based startup with a Raleigh site, is developing a gene therapy for “a monogenic disease that presents in childhood.”
- Axovant Sciences (2014) is a Swiss company that established a Durham site as it develops gene therapies to treat neurologic conditions.
- bluebird bio (1992) is a publicly held company headquartered in Cambridge, Mass., with facilities in Durham, Seattle, and Zug, Switzerland. bluebird has started European sales of LentiGlobin, a gene therapy for transfusion-dependent β-thalassemia (TDT). TDT is an inherited blood disorder that interferes with red blood cell production, leading to severe anemia. LentiGlobin is a one-time gene therapy designed to eliminate the need for blood transfusions. bluebird is also exploring LentiGlobin for addressing sickle cell disease. In 2018 bluebird received Breakthrough Therapy designation from the U.S. Food and Drug Administration for Lenti-D, its potential treatment for cerebral adrenoleukodystrophy, a rare and life-threatening neurological disorder in boys. The company’s $80 million Durham facility is a 125,000-square-foot manufacturing plant that bluebird purchased in 2017. The site’s initial build-out will allow for some 70 employees to produce clinical and commercial supplies of lentiviral vector, a critical component of the company’s gene and cell therapies. The company says it ultimately plans to have the Durham facility support all of its gene therapy programs.
- Couragen Biopharmaceutics (2012) of Chapel Hill develops gene and protein therapy products for preclinical and clinical use for the treatment of genetic and chronic diseases. Couragen also provides custom adeno-associated virus vectors as a tool for laboratory research.
- Enzerna Biosciences (2014) of Raleigh is developing molecular tools for reversible, precise manipulation of gene expression, using technologies that create sequence-specific RNA binding proteins. Enzerna, bootstrapped with a $150,000 Small Business Research Loan from NCBiotech, also offers mitochondrial toxicity models and testing.
- Genencine Therapeutics (2015) of Durham develops mRNA vaccines for the prevention and treatment of cancer, viral diseases and autoimmune diseases, as well as mRNA therapeutic drugs for genetic diseases.
- OncoTrapInc (2016) of RTP, was started by UNC professors Leaf Huang, Ph.D., and Rihe Liu, Ph.D. The company is developing a nucleic acid delivery platform to deliver therapeutic genes to tumor sites.
- Precision BioSciences (2006) of RTP utilizes a proprietary genome editing method called ARCUS to treat cancers and genetic diseasess. The company’s Manufacturing Center for Advanced Therapeutics facility produces genome-edited, off-the-shelf CAR T-cell therapy products.
- StrideBio (2015) of Durham develops engineered viral vectors for gene therapy for the treatment of rare diseases. StrideBio's technology engine utilizes structure-inspired design to engineer AAV vectors that can escape pre-existing neutralizing antibodies.
Triangle gene therapy suppliers/support companies include: