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Genzyme Licensing Gene Therapies from Biotech Start-Up

Adeno-associated virus

Adeno-associated virus (nigms.nih.gov)

11 February 2015. Voyager Therapeutics, a designer of gene therapies for central nervous system diseases, is licensing some of its treatments now in development to Genzyme Corp., a biotechnology subsidiary of the drug maker Sanofi. The deal is expected to bring the one year-old spin-off from four university research labs as much as $845 million.

Voyager Therapeutics, in Cambridge, Massachusetts, is developing treatments for debilitating 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.

The deal with Genzyme includes 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 will lead research and development of the therapies, working with Genzyme, a developer of treatments for multiple sclerosis and several rare diseases.

Genzyme will have the 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.

The agreement calls for Genzyme making an initial payment of $65 million in cash, along with a $30 million equity investment in Voyager and $5 million in other in-kind contributions. Voyager is also eligible for up to $745 million in future development and sales milestone payments, as well as royalties on sales of products from the partnership.

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. Early financing and 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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