Anyone following the market would know that companies such as Alnylam (ALNY) and ISIS (ISIS) are leading the way when it comes to moving gene silencing from the laboratory, through clinical trials and on to commercialisation. However, if all one did was follow the market then it is likely that a small, private company based in Texas would slip under the radar. That company is Gradalis®.
Gradalis has not focused on Orphan Diseases, they have gone for the big one, cancer. In partnership with the Mary Crowley Research Center they have produced outstanding results. The impact of this life changing technology has been reported in the news and the company has made a number of presentations at various conferences.
In May 2013 the company presented nine abstracts (1,2,3,4,5,6,7,8,9) at the ASGCT and it is understood that they mentioned that they are considering applying to the FDA for a Breakthrough Therapy designation for their treatment for ovarian cancer.
Gradalis currently has seven clinical trials on foot and two of them are reaching their final data collection points in the next two months. The Pll trial treating ovarian cancer reaches its final data collection point in July this year.
By any standard Gradalis is a serious player in the field of gene silencing.
So, what has this got to do with Benitec Biopharma (OTCPK:BNIKF)?
Well, Benitec is the elephant in the room for Gradalis when it comes to discussions surrounding commercialisation. The technology which Gradalis employs in its treatments uses bi-functional shrna. Benitec believes that its ddRNAi patents cover the pathway by which shrna is expressed in cells. This would cover, at least in part, Gradalis's use of bi-functional shrna, for which Gradalis has its own patents. As Gradalis is yet to take out a licence from Benitec, this represents a commercial risk to the company.
Benitec has expressed a view that it does not wish to engage in costly court battles to settle patent issues (it had a lengthy dispute with Nucleonics which it eventually won) but if Gradalis was to obtain a Breakthrough Therapy designation from the FDA with a view to commercialisation, then Benitec would have to reconsider the merits of this view. Gradalis would have to consider the implications of a possible legal challenge to its patents.
Any action in regards to patent disputes would drain both companies of potential research funds and would possibly discourage any new investors that Gradalis may need to launch its treatment commercially. None of these outcomes are particularly desirable.
Hopefully, the two companies can come to an amicable outcome without the need to engage patent attorneys.
On the positive side and regardless of any legal wrangling, over the next few months, we can expect to see some good data coming out of the Gradalis trials. This will not only validate Gradalis's outstanding work but it will also validate ddRNAi as a significant technology in the advancement of cancer treatments.
Disclosure: I am long BNIKF, .