Finally! Long overdue
(because so obvious and compelling), with the founding of Voyager Therapeutics, the
foundations have now been laid for a strong AAV (adeno-associated virus) platform gene therapy company, including DNA-directed RNAi Therapeutics. Taking advantage of new AAV shuffling approaches to
identify novel serotypes (Gao and Kay labs) to more efficiently and specifically
target cell types of interest and the world-class expertise in RNAi trigger design of two of its scientific co-founders (Kay and Zamore labs), the new company will make use of the best available
science to realize the potential of AAV and ddRNAi technology for the many diseases of
high unmet medical need in the CNS (e.g. Huntington's disease).
With $45M in start-up funding from Third Rock Ventures (the VC that has started the comparable lentiviral-based gene therapy company which recently went public, bluebird bio), they will deepen that expertise
through in-house research and
thereby sail by Benitec which sadly has refused to face the fact that in order
to be a platform company and stay relevant you need to have such efforts. If not at a time like this when interest is
high, then when?? The latest publication by Benitec and Pfizer (Denise et al. 2014) was ironically the best illustration of that omission by showing that the first-generation ddRNAi expression
technology of Benitec generates a plethora of small RNAs which obviously is less than
ideal. By contrast, work in the Kay lab
in particular (e.g. Gu et al. 2012) has shown how to generate much cleaner expression cassettes.
Voyager strutting its RNAi expertise and related therapeutic development plans is further
confirmation of the dramatic decrease in the value of Benitec's once-prominent IP position in ddRNAi (Graham patent). With an expiration of ~2018-2020 and Baulcombe the real gate-keeper (although the clock on that one is also rapidly running down),
I don’t see why there should be a need to take a license except for maybe the
likes of Calimmune which may have something close to commercialization by then
should things proceed without a hitch.
The start of a company like Voyager is always very exciting and I trust that the
scientific founders have the expertise and wisdom to guide it along the way. It is also a good time to acknowledge the
likes of Dirk Grimm (Heidelberg), Shuo Gu (NCI), and most recently Leszek Lisowski (Salk) which I had the privilege of
working next to in the Kay lab when they were critically contributing to the
technology underlying Voyager. The
pharmaceutical industry and indeed the investor community is wasting a lot of
potential by not tapping into the skills of such driven scientists just because they may not labor in one of the biotech hotbeds.
