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Start-Up Developing Gene Therapies Lands $45M in Early Funds

Adeno-associated virus

Adeno-associated virus (

12 February 2014. Voyager Therapeutics, a new start-up in Cambridge, Massachusetts creating gene therapies for disorders of the central nervous system, gained $45 million in first-round financing. Third Rock Ventures, a venture capital company specializing in life science enterprises, provided the funds for the company founded by researchers at University of Massachusetts Medical School, University of California in San Francisco, and Stanford University.

Voyager Therapeutics is developing treatments for debilitating diseases of the central nervous system including Parkinson’s disease, amyotrophic lateral sclerosis (ALS), and Friedreich’s ataxia, a rare inherited disease causing damage to the nervous system and movement problems. The company is licensing the research of the company’s founders whose work covers technologies for harnessing viruses to deliver genetic therapeutics, and treatments using RNA interference to inhibit the expression of certain genes.

The founders of Voyager Therapeutics are:

Guangping Gao, professor of microbiology at University of Massachusetts Medical School, whose work  involves the discovery, development, and use of adeno-associated viruses for gene therapy of genetic diseases. Adeno-associated viruses can infect cells, but do not integrate with the cell’s genome or cause disease, and generate a mild immune response.

Krystof Bankiewicz, professor of neurological surgery and neurology at the University of California (UC) in San Francisco, who is studying delivery of therapies to the brain using adeno-associated virus and MRI.

Mark Kay, professor of pediatrics and genetics at Stanford University, who is studying adeno-associated virus variations and structures to better understand their properties in the delivery of gene therapies.

Phillip Zamore, professor of biochemistry and molecular pharmacology at University of Massachusetts Medical School, whose work studies RNA silencing pathways and the way small RNAs inhibit the expression of genes and viruses.

Voyager is licensing research discoveries from the founders’ institutions, which include an ongoing early-stage clinical trial of a treatment for Parkinson’s disease, with colleagues at UC-San Francisco. Development of therapies for ALS and Friedreich’s ataxia are in preclinical stages. The company is also collaborating with University of Massachusetts Medical School.

Third Rock Ventures is a venture capital company specializing in life science and health care enterprises. The company is providing Voyager’s management while the company gets off the ground, including Mark Levin as interim CEO and Philip Reilly as chief medical officer.

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