Washington, D.C.-basedgene therapy outfit Regenxbio just raised $30 million to bringits platforminto the clinic.

The dollars will help Regenxbio generate clinical proof of concept data, as well as work toward in-licensing new programs. The companys also using the money to beef up its clinical and manufacturing processes, it said in a statement.

Regenxbio has developed what it calls NAV Technology a form of adeno-associated viral gene therapy that treats lysosomal storage disorders and ocular disease. Its got drugs in the pipeline that treat Hurler syndrome, Hunter syndrome, wet age-related macular degeneration and X-linked retinitis pigmentosa.

Heres how it works: In Hurler syndrome, for instance, children dont carry a gene that develops an enzyme called IDUA that breaks down complex sugars. These build up, and ultimately impairmental development, organ function, physical abilities and appearance. The NAV platform delivers a normal copy of the IDUA-producing gene which ultimately embeds itself into a patients DNA in a one-time doze so that patients can produce the enzyme. The research comes out of the University of Pennsylvania, and has been successful in vivo so its a matter of testing its efficacy in a real patient pool.

The companys also out-licensing this technology for other indications, with companies like Baxter, Dimension Therapeutics and Lysogene developing viral vector-based gene therapy for a number of other indications.

The Series C funding was led by Venrock and Brookside Capital, with other investors like Deerfield Management, FoxKiser and Fidelity Biosciences.

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Regenxbio nets $30M to bring its gene therapy to clinical trial

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