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With $45M in Funding, Startup Voyager Therapeutics Aims to Develop Expressed RNAi CNS Drugs

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Venture capital firm Third Rock Ventures last week announced that it has put up $45 million to form a new company called Voyager Therapeutics to develop adeno-associated viral vector-enabled treatments for central nervous system disorders.

Although the company's lead program is a gene-replacement therapy for Parkinson's disease, Voyager has also set its sights on using its AAV technology to develop expressed RNAi drugs — an effort that includes a preclinical program in amyotrophic lateral sclerosis, according to a company executive.

The process of establishing Voyager began a few years ago when Third Rock identified AAV-based gene therapy as a promising area of investment, given advances both in safety and delivery, Steven Paul, interim president of research and development at Voyager and a Third Rock venture partner, told Gene Silencing News.

Following meetings with key players in the field and a review of various technologies available, Third Rock decided to fully finance a $45 million Series A round to create Voyager along with four well-known researchers in the AAV and RNAi fields, he added. These include University of Massachusetts Medical School (UMMS) investigators Guangping Gao and Phillip Zamore; Mark Kay of Stanford University School of Medicine; and the University of California, San Francisco's (UCFS) Krystof Bankiewicz.

Notably, Zamore is a co-founder of Alnylam Pharmaceuticals, while Kay co-founded Avocel, an expressed RNAi startup that was acquired by Benitec Biopharma in 2004.

Thus far, little information about Voyager's core technologies and pipeline programs is being made public, including the nature of various licensing deals it has struck with UMMS, UCSF, and Stanford.

Paul confirmed that the company expects to use an RNAi approach in its ALS program, which aims to knock down the gene SOD1, and potentially with a Huntington's disease treatment, but he did not provide additional details. However, Gao has reported data based on work done in his lab on expressed RNAi in ALS.

For instance, he and his colleagues reported in 2013 on the identification of several recombinant AAV (rAAV) serotypes that could reach the spinal cord and the forebrain following intrathecal injection. One of these vectors delivered an artificial microRNA targeting SOD1 in a mouse model of ALS, which slowed disease progression.

Kay, meanwhile, has published extensively on AAV-based RNAi, including a recent paper on selecting clinically relevant rAAV vectors and a review of rAAV as a platform for RNAi therapeutics.

And Zamore has spearheaded significant research into both the basic biology of RNAi and its use as a therapeutic modality, and has collaborated with a number of different groups on using the gene-silencing technology to treat Huntington's disease.

Paul said that Voyager is not yet disclosing timelines for the clinical development of its drug candidates, which also include a treatment for the neurodegenerative disorder Friedreich's ataxia, although he noted that the Parkinson's disease therapy is currently in phase Ib testing. Such guidance, he added, will depend on "experiments and studies now underway."

Unlike some other startups, Voyager is conducting the bulk of those studies in-house, Paul said, and the company is already in the process of building out office and lab space in Cambridge, Mass.

The company has also assembled much of the team that will lead these efforts. Among them is Dinah Sah, who has assumed the role of interim senior vice president of neuroscience after spending more than five years at Alnylam Pharmaceuticals, most recently as vice president of CNS and oncology research.

Additionally, former National Institutes of Health researcher Rob Kotin has joined the company as vice president of production. Kotin is a co-inventor of the baculovirus-based AAV production system and has collaborated with investigators from the University of Iowa on treating both Huntington's disease and spinocerebellar ataxia — a condition similar to Fredreich's ataxia — using RNAi.

"Over the next three to six months, you'll be hearing more about individuals that we've recruited and hired into the company," Paul said.

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