Booming REGENXBIO Expands from 5 to 60 Employees on Strength of Gene Therapy Licenses

March 2, 2016
By Alex Keown, BioSpace.com Breaking News Staff

ROCKVILLE, Md. – Gene therapy company Regenxbio Inc. exploded onto the scene in 2015, raising more than $230 million in financing and rapidly expanding from five employees to more than 60, the Washington Business Journal reported this morning.

Regenxbio, which is developing gene therapy for the treatment of homozygous familial hypercholesterolemia, is looking to continue its momentum by filing three clinical trials by the end of this year. To support its rapid transformation, Regenxbio recently signed a lease to move from 11,000 square feet of office and lab space to more than 50,000 square feet, the Journal said. What’s driving the company’s rapid growth is the licensing of its gene therapy technology, which uses a virus to deliver healthy genes to cells in the body, to eight separate companies, including Baxalta, Inc. , Voyager Therapeutics and Dimension Therapeutics. As of Sept. 30, 2015, Regenxbio’s NAV Technology Platform is being applied in the development of 23 product candidates for a variety of diseases, including five product candidates being developed internally by Regenxbio and 18 product candidates being developed by the licensees, Regenxbio announced in November when it released third quarter financial results.

In addition to licensing its technology, Regenxbio is also using its adeno-associated viruses for its own research to target diseases that have no cure or where the fix could be caused by changes to one gene, the Journal said. Regenxbio has seen success in animal models for the treatment of homozygous familial hypercholesterolemia, a genetic defect that prevents children from clearing cholesterol from their bodies. Kenneth Mills, Regenxbio’s president and chief executive officer, told the Journal that animal models have shown the company’s gene therapy treatment, RGX-501, have shown a 90 percent reduction of cholesterol levels.

Among its targets for treatment include plans to file an Investigational New Drug application for RGX-111 for the treatment of mucopolysaccharidosis Type I in the first half of 2016, Mills said in a statement.

Although Regenxbio is looking at continued growth throughout 2016, the first month has not been kind to its stock. Since a high of $23.50 per share in December, the stock has slid to a low of $12.57 per share. Regenxbio is currently selling for $12.77 per share.

Gene therapy treatment is a high risk, high reward venture, but the promises of a therapies developed through these programs could be life changing for patients. The goal and promise of gene therapy is developing a treatment that only has to be administered once a decade or longer. Gene therapy essentially transforms cells inside a patient to harness their immune system to fight an invading disease on its own.

The gene therapy methods adopted by Regenxbio are not useful in all diseases. Mills told the Journal that AAV technology is not useful for blood diseases, such as sickle cell.