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Voyager Therapeutics, Inc. (NASDAQ: VYGR) today announced the publication in Nature Neuroscience (link to publication) of new preclinical data from ongoing efforts of Dr. Benjamin Deverman, Professor Viviana Gradinaru and the Gradinaru Laboratory at the California Institute of Technology (Caltech) to develop novel adeno-associated viral (AAV) capsids that efficiently cross the blood-brain barrier and widely transduce, or transfer, genes into the central nervous system (CNS) after intravenous administration. From these efforts, a new, second-generation AAV capsid provided up to a 100-fold increase in the transduction of the CNS in an adult preclinical model over the historical standard, AAV9, as compared with the first-generation capsid reported last year1 by the Gradinaru group that provided a more than 40-fold improvement over AAV9.

The ongoing work by Dr. Deverman, Professor Gradinaru and the Gradinaru Laboratory at Caltech continues to generate exciting AAV variant capsids for application to the treatment of CNS diseases, said Dinah Sah, Ph.D., chief scientific officer at Voyager Therapeutics. Key translational studies in non-human primates are underway by Voyager to evaluate these AAV variant capsids that have the potential to transform our ability to deliver gene therapies to the CNS. We are pleased to be partnered with the Gradinaru group at Caltech to further advance this technology.

Voyager obtained a co-exclusive license to the Caltech novel AAV capsids, intellectual property and related technology in September 2016. The license agreement covers all fields of use and includes novel AAV capsids that have demonstrated enhanced crossing of the blood-brain barrier for the potential treatment of CNS diseases following systemic administration of an AAV gene therapy vector.

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Voyager Therapeutics (VYGR) Reports Publication of 2nd ... - StreetInsider.com