Taysha is joining the fast-growing community of cutting-edge gene- and cell-therapy companies setting up shop in the Research Triangle, where decades of investment and workforce training have created a magnet for the discovery and manufacture of science’s game-changers in fighting some of humankind’s most fearsome maladies.
The company is developing gene therapies that use benign adeno-associated viruses (AAV) as “vectors,” or carriers, to transport genetic corrections to otherwise defective areas of the body. Taysha is initially targeting genetic diseases of the central nervous system, such as CLN1 disease, also called infantile Batten disease, which causes developmental delays in children, and Rett syndrome, a rare genetic mutation affecting brain development in young girls.
Taysha has a partnership with the University of Texas Southwestern Medical Center near its Dallas headquarters that accommodates some initial production of its gene therapies. And the company announced a partnership last month to add manufacturing capacity at therapeutics developer Catalent’s Maryland-based gene therapy facilities. But the RTP investment is aimed at large-scale manufacturing of Taysha’s product line as it evolves.
There are reasons the Research Triangle has become an epicenter for AAV technology, used by most gene therapy companies today. It was developed by Jude Samulski, Ph.D., of Chapel Hill, who holds the first U.S. patent for inserting non-AAV genes into AAV. Samulski 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 led UNC’s Gene Therapy Center for several years and in 2001 co-founded Asklepios BioPharmaceutical (AskBio) in RTP, which Bayer recently bought for $4 billion.
AskBio itself spun out four gene therapy startups in recent years: NanoCor Therapeutics, Chatham Therapeutics, Bamboo Therapeutics and Actus Therapeutics. Chatham was acquired by Takeda, and Bamboo was acquired by Pfizer.
That base of gene therapy science, coupled with North Carolina’s storied life sciences workforce development system and positive business climate, have drawn billions of dollars of investment from gene and cell therapy companies to the Triangle in recent years.
Taysha has had numerous connections with North Carolina’s gene therapy community. One of the company’s founders, early chief scientific officer Steven Gray, worked in Samulski’s lab at UNC. Also, several other members of the management team came from AveXis. Swiss drugmaker Novartis bought AveXis for $8.7 billion in 2018 and renamed it Novartis Gene Therapies.
“Taysha is all about helping patients, and this investment underlines North Carolina’s commitment to help the company achieve that vision,” said Bill Bullock, senior vice president of economic development and statewide operations for the North Carolina Biotechnology Center, a partner in Taysha’s recruitment to the state.
“It also reinforces North Carolina’s commitment to providing best-in-class talent to attract these types of investments, and the state’s position as a global leader in gene therapy manufacturing.”
Taysha is a publicly held company trading on the Nasdaq exchange with the symbol TSHA. The company says Taysha is a word in the Caddo Native American language meaning “ally” or “friend.”
(C) N.C. Biotech Center