Kite Pharma, a Gilead company, and Sangamo Therapeutics announced a worldwide collaboration worth a potential $3B to use Sangamo’s zinc finger nuclease (ZFN) technology platform to develop next-generation cell-based therapeutics for oncology.
Kite plans to apply the technology to both allogeneic cell therapies and renewable stem cells, with the most obvious application being the enhancement of the antitumor activity of Kite’s CAR-T candidates via deleting immune checkpoint molecules such as PD-1, which can be used by tumours to dampen the immune response. Although ZFN is an older version of CRISPR, by choosing this platform, Kite is mitigating the risk for unexpected safety signals, and most importantly, could be saving itself from having issues with intellectual property (IP) rights in the future.
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CRISPR is seen as a next-generation improvement on ZFN technology, as CRISPR sequences are comparatively much easier, cheaper, and faster to design and produce. Although ZFN does have its advantages, such as lower potential levels of immunogenicity and ease of in vivo delivery due to the small size of the required gene expression cassettes, most gene-editing biotech companies have chosen to focus on CRISPR technology.
Editas Medicine, Intellia Therapeutics, and CRISPR Therapeutics are just three of the companies that have entered this arena in recent years, and all three have preclinical programs aimed at developing CRISPR for oncology. Compared to ZFN, however, CRISPR is in its infancy from a clinical perspective. The first human trial of CRISPR was only launched in China in 2016, and the first trial in the US has not yet begun. In contrast, Sangamo has treated over 100 patients in early-stage clinical trials and found no significant safety issues thus far. Given the fact that CAR-T carries the risk of severe and potentially lethal toxicity, by choosing a gene-editing technology with a known safety profile, Kite can minimize the risk of additive toxicity.
Perhaps an even bigger concern than the lack of in-human clinical trial data for CRISPR is the IP issue surrounding the technology. A bitter battle over patent rights to the CRISPR-Cas9 technology has been raging in US and European courts, with both the Broad Institute and the University of California claiming inventorship over the related patents. In contrast, Sangamo is the primary patent holder for its ZFN platform, as the company has worked on the technology for over 20 years. If Kite were to market a cell-based therapy that used CRISPR technology, it is likely that IP rights and hefty patent licensing fees would have to be negotiated with the patent holders, whereas with the ZFN technology, no such problems exist.
Despite scepticism over why Kite has chosen a relatively outdated technology versus a newer, more exciting version, this could prove to be a good move from a commercial perspective and may spur other companies to take a second look at alternative gene-modification strategies.
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By GlobalDataRelated Reports
GlobalData (2016). Expert Insight: CRISPR development race has “no clear winner” despite China trial approval, steep hills en route to market, Aug 2016
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