Durham County, NC — A 2-year-old suburban Chicago gene therapy company, Jaguar Gene Therapy, LLC, announced plans to invest $125.4 million and create 200 jobs between 2024 and 2028 at a new Research Triangle Park manufacturing facility.
The fast-growing young company plans to manufacture clinical and commercial quantities of its novel adeno-associated virus (AAV)-based gene therapies in the Durham County factory at 14 T.W. Alexander Drive in RTP. It intends to pay an average salary of $92,530, compared to the county’s average of $75,892. The facility is projected to increase the state’s gross domestic product by more than $279 million when fully operational, and increase net state revenue by $35 million.
AAVs are small viruses that can infect cells without causing any known harm, making them ideal vectors, or carriers, of genetic material into cells. The gene therapy technology was developed by Triangle scientist and entrepreneur Jude Samulski, who holds the first U.S. patent for inserting non-AAV genes into AAV. He is the lead inventor on more than 300 patents in the field of AAV vectors and gene therapy.
Samulski was recruited to the University of North Carolina School of Medicine in 1993 with nearly $250,000 in grant funding from the North Carolina Biotechnology Center. He has led UNC’s Gene Therapy Center for several years and co-founded Asklepios BioPharmaceutical (AskBio) and numerous spinouts that have resulted in billions of dollars of mergers and acquisitions.
“North Carolina’s diverse life sciences presence continues to attract innovative biopharmaceutical companies,” said Governor Roy Cooper, in a news release announcing the project. “Jaguar’s expansion into biomanufacturing in Research Triangle Park, together with its process sciences laboratory in Cary, reinforces North Carolina’s reputation as a premier location for these companies.”
The company already has 25 employees in the Triangle.
Jaguar was formed in 2019 by a group of former AveXis leaders, so they’re familiar with the Research Triangle area’s growing prominence in gene and cell therapies. AveXis, which made gene therapies at a fast-growing Durham County facility, was purchased in 2018 for $8.7 billion by the Swiss drugmaker Novartis. So that company is now named Novartis Gene Therapies.
“Jaguar Gene Therapy belongs in North Carolina’s growing cell- and gene-therapy ecosystem,” said Bill Bullock, senior vice president for economic development and statewide operations at the North Carolina Biotechnology Center, who helped support the company’s selection of North Carolina for this project. NCBiotech is adding a $100,000 award to other state and local incentives undergirding the company’s choice of North Carolina over sites it had considered in Columbus, Ohio and Indianapolis. State officials earlier today agreed to provide Jaguar a total of $3.14 million in Job Development Investment Grant funding. That was also bolstered by commitments of $500,000 from the community college system for worker training, and $525,000 from Durham County.
“Jaguar’s leaders know the long-term commitments required to bring great ideas to the marketplace. And they recognize that North Carolina has been making a well-focused investment in workforce preparation, breakthrough research and business development, all within a unique environment of collaboration. NCBiotech is honored to have contributed to the partnership that led Jaguar Gene Therapy here.”
“We believe there is significant value in owning the capability to manufacture multiple gene therapies from process development to commercial GMP scale,” said Joe Nolan, CEO of Jaguar Gene Therapy. “This strategic investment will leverage the team’s proven CMC (Chemistry, Manufacturing and Controls) expertise and may enable Jaguar to potentially accelerate development timelines and bring our therapies to patients and families as safely and as rapidly as possible.”
Jaguar plans to use the latest technologies and purification techniques at the new 147,000-square-foot facility to increase product yields while reducing product impurities. The team’s experience in developing a scalable and commercially viable manufacturing process at the outset has guided the company’s approach to invest early and often in manufacturing. In doing so, the company aims to be on the leading edge of producing the purest and most potent gene therapy products.
Besides NCBiotech, the N.C. Department of Commerce and the Economic Development Partnership of North Carolina, other key partners in the project include the North Carolina General Assembly, North Carolina Community College System, Durham County, the Greater Durham Chamber of Commerce and Duke Energy.
“Our new GMP facility represents a major step forward for Jaguar in our mission to accelerate gene therapy breakthroughs in patient populations with large unmet need,” said Andrew Knudten, M.S., MBA, chief operating officer of Jaguar Gene Therapy. “North Carolina is known for its innovation in biotechnology and its highly talented workforce, both of which are crucial as we strive to raise the bar for manufacturing safe and effective gene therapies.”
“With a strong roster of globally recognized biomanufacturers and gene and cell therapy leaders, North Carolina is a top choice for growing biotech companies,” said North Carolina Commerce Secretary Machelle Baker Sanders. “North Carolina has the world-renowned researchers, Tier 1 universities and strategic partnerships to maintain our thriving life sciences ecosystem. With our First in Talent plan, workforce training will continue to develop the diverse and highly-skilled talent for the life changing work that companies like Jaguar will create both now and in the future.”
Jaguar’s initial pre-clinical pipeline of AAV9-based gene therapies under development includes:
JAG101, a treatment for galactosemia, a metabolic condition that in its most severe form affects an estimated 4,500 patients in the United States. An additional estimated 17,000 individuals in the United States have a less-severe form of the disease yet still suffer from long-term effects. Galactosemia is diagnosed within months of birth and is caused by an inborn error of carbohydrate metabolism, which impairs the body’s ability to process and produce energy from galactose, one of the sugars in breast milk and formula. Consequences of the disease can include cataracts, liver failure, kidney dysfunction and brain damage (speech abnormalities). Because of its severity, galactosemia has already been added as part of newborn screening in the United States and in other global markets. The current standard of care is a strict diet that has modest effects in some patients but is often not sufficient to prevent long-term complications.
JAG201, a treatment for a specific genetic cause of autism spectrum disorder. Hallmarks of autism disorders include seizures, emotional/social interaction issues, and restricted and repetitive behaviors that can persist and interfere with everyday life. There are currently no treatment options available for the estimated 30,000 patients in the United States with the genetically caused autism spectrum disorder that Jaguar is targeting.
JAG301, a treatment for Type 1 diabetes, a metabolic autoimmune disease that currently requires lifelong insulin injection dependency. Serious complications from Type 1 diabetes can include frequent hospitalizations, blindness, heart disease, stroke, kidney damage and nerve damage. Jaguar is evaluating proof-of-concept data to best determine the appropriate clinically relevant patients within the newly diagnosed population.
Additionally, Axovia Therapeutics, a British majority-owned Jaguar subsidiary that is focused on creating gene therapies for ciliopathies — complex disorders caused by genetic mutations which result in defective or dysfunctional hairlike structures called cilia in many organs of the human body. Ciliopathies include such disorders as polycystic kidney disease and retinitis pigmentosa. Avoxia is advancing AXV101. That’s a treatment for BBS1, a subset of Bardet-Biedl syndrome (BBS). A life-threatening neurometabolic condition, BBS causes progressive vision loss, severe obesity, learning disorders and kidney disease.
In mid-April Jaguar closed a $139 million Series B funding round co-led by Eli Lilly and Company and Deerfield Management. Also participating in the round were ARCH Venture Partners, co-founded by Robert Nelsen and one of the largest early-stage technology venture firms in the United States, Goldman Sachs, and Nolan Capital, the investment fund of former AveXis CEO and current Jaguar Executive Chairman Sean P. Nolan.