Gene therapy pioneer ReGenX fuels R&D, hiring spree with $30M VC round

Cashing in on the current enthusiasm in venture circles for gene therapy upstarts, ReGenX has snagged a $30 million round that will finance a round of new hires as it sets sail for the clinic with lead programs for rare diseases of the central nervous system.

While keeping a low profile since its launch in 2009 with gene therapy technology originally sourced from the academic visionary James Wilson at Penn, ReGenX became a prime source for the AAV vectors needed to deliver corrective genes that are being used to fix a whole slate of ailments. The company spun out Dimension and executed tech licensing deals with a lineup that includes Third Rock's Voyager, Lysogene and AAVLife.

Now it wants to do its own clinical development work. And it has the money needed to give it a serious shot.

"I think we have a runway of up to 2 years, depending on how aggressive we get with other opportunities," CEO Ken Mills tells FierceBiotech. "We may in-license other opportunities or differentiate a little bit. For me the $30 million is a first step, it sets a new plateau to build off of."

Venrock and Brookside Capital led the round, with Deerfield Management and an unnamed investor joining in with the biotech's two startup backers: Fidelity and FoxKiser.

The Washington, DC-based developer got started with a virtual staff and now has 10 employees on the payroll, including their new chief medical officer. By the end of this year, Mills plans to have a staff of about 30 working in Maryland's tech corridor, where he's about to ink a deal for a new home office. The beefed up staff will be tasked with development, expanding manufacturing as well as commercial and business development work.

Like others in the re-emerging gene therapy field, ReGenX is working to get into the clinic with treatments for rare diseases. The biotech is focused on CNS diseases like Hurler Syndrome (MPS I) and Hunter Syndrome (MPS II). Last summer the company successfully concluded a proof-of-concept animal study for a gene therapy that addresses a rare lysosomal storage disorder.

ReGenX's scientific godfather James Wilson--who signed on as a scientific adviser last fall--was there at the dawn of gene therapy work, saw it crash and burn over lethal safety issues years ago and witnessed its recent resurrection as a whole new generation of companies set out to prove that the technology's time has arrived. But Wilson--who headed to San Francisco to talk up ReGenX at the JP Morgan conference last week--also has a keen sense of just how risky the renaissant field remains, despite the flurry of new deals and string of newly minted public companies that has emerged.

"I would never have predicted what's happened in the last 18 months," Wilson told The New York Times' Andrew Pollack, while underscoring the technology challenges that remained. "We're in a bubble, and gene therapy is at the center of it. One learns humility. That's what I learned."

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