- The European Patent Office plans to grant a broad patent covering CRISPR/cas9 gene-editing technology to the group of claimants affiliated with the University of California (UC), just six weeks after the U.S. Patent Trial and Appeal Board (PTAB) sided with the competing Broad Institute of MIT and Harvard over similar claims in the U.S.
- The EPO's decision is welcome news for CRISPR Therapeutics and ERS Genomics, two biotechs which license the hot technology from Emmanuelle Charpentier and UC.
- CRISPR Therapeutics, which announced the news of the EPO's intention to grant a patent Tuesday morning, believes the patent reflects "the broad applicability" of the company's foundational intellectual property. Securing a patent in Europe could help CRISPR stake out a strong blocking position that will help in commercialization efforts down the road.
Despite the attention paid to the PTAB's decision last month, the legal maneuvering to secure a dominant position in the emerging field is far from over.
The highly technical patent dispute has been so closely followed because of the vast potential of CRISPR gene-editing technology to push the boundaries of what's possible in a wide array of therapeutic areas, as well as in agriculture and industrial biotechnology.
With the EPO now weighing in, UC's camp appears to hold an upper hand in Europe while the Broad Institute has won the first round in the U.S. CRISPR Therapeutics and ERS Genomics license the technology from Charpentier and UC, while Editas Medicine licenses from the Broad.
The patent set to be granted by the EPO would cover use of a CRISPR/cas9 single-guide system in cellular and non-cellular settings, importantly encompassing both human and animal cells as well as claims for therapeutic use.
In ruling in favor of UC and Emmanuelle Charpentier, the EPO dismissed a number of so-called "third-party observations" — including those filed by the Broad Institute — that sought to prevent or delay a patent grant.
For CRISPR, the patent award to the UC side could help it create a blocking IP position in the EU, strengthening its market position for commercialization of future CRISPR-based therapeutics in that jurisdiction. The biotech's most advanced pipeline projects are currently in beta-thalassemia and sickle cell disease (in collaboration with Vertex Pharmaceuticals), with an array of other programs covering gene editing of hematopoietic cells and in vivo targeting in the liver.
Typically, the U.S. is a more lucrative market than Europe for most major indications. But CRISPR Chief Business Officer Sam Kulkarni, reached by phone after the announcement, said the market opportunity for CRISPR-based therapeutics could potentially be equal or greater in Europe for the indications CRISPR plans to target. In beta-thalassemia, for example, CRISPR plans to start its clinical trials in Europe first, Kulkarni said.
In the U.S., the PTAB's ruling last month effectively supported patents already awarded to the Broad Institute, judging claims by UC to be separately patentable and therefore not overlapping (or "interfering" in the language of patent law). While a setback, UC and CRISPR believe that this now allows the U.S. Patent Office to review UC's original patent application, potentially leading to a separate patent grant covering CRISPR use in all cells.
That would lead to a seemingly contradictory result: UC with a patent on use of CRISPR in all cells and the Broad with a patent for use of CRISPR specifically in eukaryotic cells.
UC and CRISPR could also appeal the PTAB's ruling, which would pause review of that specific patent application. Kulkarni said the two are still considering options but that there are sufficient grounds to appeal.
But, even in that scenario, the duo can simultaneously move forward with other patent applications to try to strengthen their IP position vis a vis the Broad and Editas.
While markets have closely followed each patent judgment, the competing awards and claims could eventually lead to a situation where the biotechs on each side cross-license use of CRISPR technology. Such a development would likely not impede pipeline development or growth however, executives at CRISPR Therapeutics had said in previous interviews.
Shares in CRISPR rose more nearly 10% by Tuesday afternoon. Somewhat counter-intuitively, Editas Medicine stock also jumped in value, rising a similar 6% early before falling back to a more modest 2% gain.