By Callum Borchers/Globe Staff/February 12, 2014

A Cambridge biotechnology company launching Wednesday is taking aim at Parkinsons disease and ALS with a new gene therapy that deliberately infects patients with a virus.

The firm, Voyager Therapeutics, plans to use a class of viruses known as adeno-associated viruses as carriers to deliver vital proteins to the brain. Intentional infection may be counterintuitive, but the viruses used in the therapy are harmless to humans, making them ideal vehicles for moving proteins throughout the body, without troublesome side effects.

Boston venture capital firm Third Rock Ventures considered Voyagers research so promising that it invested $45 million to get the company off the ground, an unusually big bet on such an early stage life sciences firm.

Were just convinced that these viruses are going to be incredibly important delivery vehicles to different parts of the body and make a big difference in a lot of very serious disorders, said Third Rock cofounder Mark Levin, who will serve as Voyagers interim chief executive during the companys start-up phase.

The investment in Voyager marks Third Rocks latest foray into genetic medicine and the treatment of rare diseases. Bluebird bio of Cambridge, another gene therapy company in its portfolio, raised more than $100 million in an initial public stock offering last June. Bluebird is working on a treatment to slow the progression of a genetic brain disorder called childhood cerebral adrenoleukodystrophy, or CCALD.

In November, Third Rock joined two other venture firms in putting a combined $43 million behind a Cambridge start-up called Editas Medicine, which is developing a technique to edit faulty genes, such as those that cause Huntingtons disease and sickle cell anemia.

The investments reflect a broader belief among the scientific community that gene therapy could be the key to effectively treating some of the worlds most challenging disorders. Gene therapy techniques typically involve replacing a mutated gene with a healthy version or turning off a gene that causes disease.

Voyager plans to use adeno-associated viruses as carriers for both techniques. To treat Parkinsons, for instance, Voyager will use viruses to deliver a missing protein. For ALS, the viruses will help shut down a harmful protein.

Expecting gene therapy to produce cures for rare diseases might be unrealistic, Levin said, but the idea is to make a dramatic difference in patients lives.

See the article here:
Biotech start-up Voyager Therapeutics uses new gene therapy to attack diseases - Boston.com

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