Gene therapy pioneer ReGenX raises $70.5M to go it alone

Read enough about gene therapy's recent renaissance and you're bound to come across one company over and over. Rockville, MD's ReGenX Bio has signed 8 out-licensing deals tied to its in-house approach to getting corrective genes to their targets, providing the engine behind headline-grabbing projects from Voyager Therapeutics, Dimension Therapeutics and others.

Now, after years of lending out its platform, ReGenX is turning its attention to a proprietary pipeline of gene therapies, hauling in a $70.5 million D round to get its top prospects into clinical trials.

The latest round, led by new investor Vivo Capital and existing investors Venrock and Brookside Capital, will help ReGenX flesh out its staff, build up its infrastructure and evolve into a clinical-stage biotech, CEO Ken Mills said.

The company's product engine, called NAV, uses harmless adeno-associated viruses (AAV) to deliver functional genes that can replace or knock down the errant ones that lead to diseases. ReGenX's NAV-powered pipeline includes preclinical treatments for the rare CNS disorders Hurler syndrome and Hunter syndrome, plus a one-time treatment for wet age-related macular degeneration, the most common cause of blindness among the elderly.

The plan, Mills said, is to push those candidates toward clinical trials this year.

In the meantime, ReGenX is busy building out its team. In January, when the biotech closed a $30 million, it had a staff of 10. Now it's working with 18 people and continuing to recruit, Mills said, looking to expand its brain trust of clinical development experts on the way to Phase I.

Assembling the Series D--which looped in a wide range of investors including Fidelity Biosciences, Foresite Capital Management and Janus Capital Management--ReGenX never discussed the idea of looking for a buyer in the near term, Mills said.

"We want to be a great company and a long-term company and bring multiple products to patients," Mills said. "We want to be here in 10 years."

- read the statement (PDF)

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