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Gene Therapy Biotech Raises $60M in Venture Funds

Adeno-associated virus

Adeno-associated virus (

13 April 2015. Voyager Therapeutics, a biotechnology company developing gene therapies for central nervous system disorders, raised $60 million in its second venture financing round. Financing for the 1 year-old enterprise in Cambridge, Massachusetts was led by new investors Brookside Capital and Partner Fund Management, with participation by Wellington Management Company and Casdin Capital, also new investors, as well as other investors who were not disclosed.

Voyager is developing treatments for diseases of the central nervous system including Parkinson’s disease, amyotrophic lateral sclerosis (ALS), Friedreich’s ataxia, a rare inherited disease causing damage to the nervous system and movement problems, and Huntington’s disease, an inherited brain disorder that results in progressive loss of both mental faculties and physical control. The company is commercializing research by 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.

Voyager’s technology harnesses adeno-associated viruses to deliver healthy genetic material for expressing proteins missing from the mutated or damaged genes causing the disorder. Adeno-associated viruses are benign, naturally occurring microbes that can infect cells, but do not integrate with the cell’s genome or cause disease, and generate a mild immune response. One of the company’s founders is 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 inherited diseases.

In February 2015, Voyager announced a licensing deal with Genzyme, a biotechnology subsidiary of the drug maker Sanofi, that could earn Voyager as much $845 million. The agreement covers several Voyager gene therapies for Parkinson’s disease, Friedreich’s ataxia and Huntington’s disease, as well as other unspecified disorders of the central nervous system. Voyager is leading research and development of the therapies, working with Genzyme, a developer of treatments for multiple sclerosis and several rare diseases.

Genzyme has an option to license further rights to the therapies following early clinical trials. Voyager will continue to hold the U.S. rights to its lead treatments being developed for Parkinson’s disease and Friedreich’s ataxia, and will split profits in the U.S. with Genzyme from its Huntington’s disease therapy. Voyager’s ALS treatment is not included in the collaboration.

In addition to Guangping Gao, Voyager was founded by three other medical, genetics, and pharmacology faculty from University of Massachusetts, University of California in San Francisco, and Stanford University. First round financing of $45 million and early management for Voyager was provided by Third Rock Ventures, a life sciences venture capital company.

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Hat tip: Fortune/Term Sheet

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