After a string of gene therapy licensing deals, ReGenX says it's time to shine

ReGenX Biosciences is one of the most intriguing gene therapy biotechs you never heard of. And now the executive team in charge intends to start changing that.

Born in 2009 with a package of gene therapy technology developed by Penn's pioneering James Wilson and outlicensed by GlaxoSmithKline ($GSK), the biotech has parlayed its trove of vectors into a string of deals with startups like Third Rock's Voyager, Lysogene, and AAVLife, and spun out Dimension, which raised a startup round from the deep-pocket players at Fidelity.

Today the Washington D.C.-based company is talking up an early stage but important inflection point for the company. It successfully concluded a proof-of-concept animal study for a gene therapy that addresses a rare lysosomal storage disorder. Now it is working on raising a new venture round that will allow the company to complete an IND and then jumping into the clinic with its own program in 2016.

The animal study focused on mucopolysaccharidosis type I (MPS I), a lysosomal storage disease also called Hurler sydrome which is caused by the body's inability to produce the α-L-iduronidase or (IDUA) enzyme. Using a cat model for the disease, researchers at the Perelman School of Medicine at the University of Pennsylvania were able to boost levels of IDUA after a solo injection of AAV9 expressing the IDUA gene, "restoring to normal the histological and biochemical features of MPS I in the CNS."

This isn't a new field. Genzyme and BioMarin ($BMRN) are well into the enzyme replacement therapy field already. But their drugs are delivered systemically through an IV, says ReGenX CEO Ken Mills, and miss the neurological symptoms of the disease that can be addressed by gene therapy.

"We are trying to address the unmet need, using the vector to deliver a healthy copy of the missing gene to the brain, arresting these symptoms," says Mills.

In most cases, successful animal studies are generally an invitation to begin a very long and very risky march through the clinic. But gene therapy is a little different, in the sense that early data from even a handful of human patients can provide key insights into whether a new treatment can work. ReGenX's team has begun to see firsthand what its technology can do at other companies, and now they believe they're ready to build a pipeline of their own clinical-stage efforts.

To be sure, there are some risky steps ahead for ReGenX, which has a virtual staff of 7. But with gene therapy proving one of the hottest fields in biotech right now, and a scientific founder who's a high-profile figure as head of the gene therapy program at Penn, ReGenX says it's time for the company to take its place alongside other prominent players.

While Fidelity was helping launch Dimension, it also participated in an $8 million raise with ReGenX's original investors, a group credited with about half of the $30 million raised to date. Now the CEO says that they intend to raise more than the investors have put in so far.

It's time to take the next step.

- here's the release