Third Rock puts up $45M for a voyage into gene therapy

Third Rock Ventures co-founder Mark Levin

Third Rock Ventures has cut the ribbon on its latest startup, pledging $45 million to launch Cambridge, MA's Voyager Therapeutics, a gene therapy biotech with a promising platform for central nervous system treatments.

Voyager's approach to the field involves hitching a therapeutic payload 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 said. The company is getting off the ground with a Phase Ib treatment for Parkinson's disease and two preclinical assets with applications in Friedreich's ataxia and amyotrophic lateral sclerosis, or Lou Gehrig's disease.

As is common for Third Rock startups, co-founder Mark Levin will begin at the helm as Voyager's interim CEO, and the company's $45 million seed round should carry it for three years, he said. By then, Levin expects Voyager to have multiple compounds in the clinic, a few weighty partnerships in place and, perhaps most important, a well-fleshed-out platform for developing new medicines.

Voyager is Levin and Third Rock's second major bet on gene therapy, following its success with the recently IPO'd bluebird bio ($BLUE), which uses lentivirus to deliver corrective payloads. Their work with that company gave Levin and his team an affinity for the field, and, wooed by some tantalizing safety and efficacy data on AAV as a delivery vector, they decided to get back in the game.

"What we realized was that there wasn't a broad product engine in AAV," Levin said. So Voyager is building one, assembling all of the virus' naturally occurring serotypes to "mix and match" them, he said, "which will create an even safer virus, and then that will create a whole new generation of intellectual property."

The company has recruited AAV and RNAi experts from the University of Massachusetts Medical School, Stanford and the University of California, San Francisco, in-licensing the technology it needs to move the needle in gene therapy, Levin said. Eventually, Voyager's platform will allow for applications in Alzheimer's disease, Huntington's disease and other fatal or debilitating CNS disorders, he said.

Meanwhile, between bluebird, uniQure ($QURE) and a bevy of similar startups attracting venture attention, gene therapy seems to be having a bit of a second honeymoon in the biotech world. But the seemingly faddish excitement is just another case of science moving slower than hype, Levin said, as it has taken nearly 25 years for researchers to hammer out issues of safety and deliverability. Now, after numerous advances in design and development, gene therapy is poised to change the standard of care for some hard-to-treat diseases, Levin said, and Voyager plans to play a leading role in the emerging field.

"We really think we can make a big difference for patients over the next many years," he said. "We have a chance to revolutionize CNS therapy by going after very specific proteins in the brain."

- read the announcement

Special Report: The Top 15 Biotech VC firms - Third Rock Ventures

Suggested Articles

Fifteen of the 22 patients in a gene therapy trial no longer needed transfusions, while the remainder needed fewer transfusions.

Argos Therapeutics is ending its kidney cancer trial and mulling options, including a merger or sale, to stay alive.

CNS Pharma says berubicin is the first anthracycline drug to cross the blood-brain barrier and could transform treatment of the highly invasive brain tumor.