Voyager Therapeutics - 2014 Fierce 15

Voyager Therapeutics
Voyager sets out to change the game in gene therapy

CEO: Steven Paul
Based: Cambridge, MA
Founded: 2014
Clinical focus: Gene therapy

The scoop: Gene therapy has been through the requisite ringer for new technologies in biotech, first billed as a panacea, then castigated as overhyped, and, now, enjoying a second honeymoon among investors and investigators. Voyager Therapeutics has positioned itself to stand out among the many startups toiling in the space, at once working on near-term treatments for genetic diseases and investing in next-generation approaches to creating one-time treatments. Now, with $45 million courtesy of Third Rock Ventures and a Big Pharma R&D veteran at the helm, Voyager has its sights set on disorders of the central nervous system.

What makes Voyager Fierce: Like the majority of Third Rock's seedlings, Voyager fancies itself less as a candidate-driven biotech and more as a product engine, and the company is employing a bifurcated business model in its quest to develop new CNS treatments.

Voyager's immediate plans involve hitching therapeutic payloads to an adeno-associated virus (AAV), allowing it to safely and precisely deliver either a replacement healthy gene or a knock-down blow to a mutated one. The company is getting off the ground with a Phase Ib study on a therapy for Parkinson's disease that could make a world of difference for patients who don't respond to standard treatment, CEO Steven Paul said. The old drugs levodopa and carbidopa have long proven themselves effective in treating PD symptoms, but the long-generic treatments don't work for everyone. Voyager's candidate, for which early results are expected next year, has the promise of not only reversing nonresponse but also lowering the doses patients need to see results, Paul said.

Beyond that, Voyager is working up preclinical AAV-powered treatments for Friedreich's ataxia, a rare neurodegenerative ailment, and ALS (the company took the Ice Bucket Challenge, Paul is quick to point out).

But Voyager's greatest promise may lie in some work even farther from the clinic. In tandem with its near-term drug development priorities, the nascent biotech is working on a from-scratch approach to delivering one-time treatments, bringing in AAV and RNAi experts from the University of Massachusetts Medical School, Stanford and the University of California, San Francisco.

"What we're doing is designing new vectors," Paul said. "Others are using existing ones--and we are, too, because we need to get started--but we believe that years from now we'll be able to engineer much better ones than currently exist, better at crossing the blood-brain barrier, better at using the cerebrospinal fluid."

To get there, Voyager is gathering all of AAV's available serotypes and mixing and matching them in hopes of developing safer, more predictable delivery vehicles. If successful, the company could be sitting on the key to future treatments for Alzheimer's disease, Huntington's disease and non-CNS ailments.

"Once you get a vector that's suitable for, say, delivery to the brain, you can design these 'drugs' pretty quickly and pretty efficiently," Paul said.

And Paul, after 37 years at NIH and Eli Lilly ($LLY), is embarking on a third career in the fast-paced world of biotech.

In 2010, he retired as president of the Lilly Research Laboratories and joined up with Third Rock as a venture partner, helping found the recently IPO'd CNS developer Sage Therapeutics ($SAGE) and settling into a more hands-off role. However, as Voyager began to take shape, the R&D veteran found himself allured by the science, the technology and the growing team of experts.

"It just occurred to me as we were out looking at CEOs: Maybe this is something I could do and do pretty well," Paul said. At Lilly, he managed a $4.5-billion-a-year budget, steering the efforts of about 8,000 researchers, and while that was rewarding, in biotech "you get closer to the science; you get closer to the real key decisions in many respects," he said.

Investor: Third Rock Ventures

For more:
Gene therapy biotech Voyager snags a former Lilly exec to take the reins
Third Rock puts up $45M for a voyage into gene therapy

-- Damian Garde (email | Twitter)

Voyager Therapeutics - 2014 Fierce 15