CRISPR Patent Ruling Lifts Editas, Disappoints Intellia and CRISPR Therapeutics
US Decision Goes Against Nobel Prize Winners
Executive Summary
A US decision to grant a CRISPR Cas9 patent to The Broad Institute lifts Editas but adds uncertainty to the IP estate of Intellia and CRISPR Therapeutics.
A long-running legal battle over patents for CRISPR Cas9 gene editing has seen the US Patent Office’s Patent Trial and Appeal Board rule against the two scientists credited with discovering the technology, Jennifer Doudna of the University of California and the University of Vienna’s Emmanuelle Charpentier (collectively known as CVC), and in favor of Feng Zhang of the Broad Institute of MIT and Harvard.
Charpentier and Doudna were awarded the Nobel Prize in Chemistry two years ago for the discovery of the bacteria-derived gene-editing technique. However, the US Patent Office found that it was Zhang who first used the technique in eukaryotic cells including animal and human cells and therefore should hold the specific patents for CRISPR/Cas9 editing in human cells, including No. 8,697,359.
The patent office’s opinion was based on scientific records showing the problems the CVC labs had had in getting their invention to work in animal cells (starting in zebrafish) and ruled that the Broad Institute made this breakthrough first. The dispute first began when CVC brought a case against the Broad for “patent interference” in 2017, but the decision brings the matter to a close, for now at least.
The ruling has implications for the many biopharma companies now using the CRISPR-Cas9 technology to develop new medicines, and could give Editas Medicines an advantage in the field, as it had licensed its technology from the Broad Institute.
It will be less welcomed by a group of biotech companies who were founded on the now-invalidated IP established by Charpentier and Doudna: CRISPR Therapeutics, Caribou Biosciences and ERS Genomics and Intellia Therapeutics. (Also see "New Data Boost For Intellia’s Pioneering In Vivo Gene-Editing Therapy" - Scrip, 1 Mar, 2022.)
News of the decision on 28 February was particularly bad timing for Intellia, as earlier in the day it had unveiled data on what could become the first ever in vivo gene editing therapy, NTLA-2001.
Intellia saw its shares fall 9.2% in after-hours trading, while CRISPR Therapeutics dropped 1.4%. By contrast, Editas saw its share price rise 10% to $18.90.
James Mullen
Editas CEO James Mullen said in a statement that both academic groups had made important contributions to the science that ultimately made gene-editing a reality, but that the decision “reaffirms the strength of our foundational intellectual property.”
The news is a tonic for the company, which has seen investor confidence decline after a string of senior management departures. The latest of these came last month when chief medical officer Lisa Michaels was dismissed without explanation.
The Cambridge, MA-based biotech presented initial data on its rare blindness therapy EDIT-101 first human clinical trials last year, but a sparsity of data left investors skeptical. (Also see "First Results From Editas CRISPR Eye Therapy Underwhelm Investors" - Scrip, 30 Sep, 2021.)
The latest ruling is not necessarily the end of the dispute, however, as the losing side could appeal to the federal circuit court. The University of California said it believed the PTAB had made a number of errors and that CVC was considering various options to challenge the decision.
The Broad said in a statement that it had been calling for a joint licensing strategy, or patent pools, for more than eight years, even before patents were issued to it or the University of California-Berkeley (UCB). It said the goal was with ensuring “open, equitable, and streamlined access to these transformative tools” and said it was optimistic this could be achieved.
Intellia and CRISPR Therapeutics are reviewing the ruling, and could also appeal. The patent rights are much clearer outside the US, as CVC is recognized as the foundational patent holder.
Analysts at JMP said the decision was unlikely to be very grave for these two companies, as their patent estates go far beyond the Cas9 application patent that is being disputed.
While the uncertainty around the decisions and implications will continue, the worst-case scenario for the companies will be that they must license the technology again from the Broad.
Among the rivals, CRISPR Therapeutics looks set to have the first regulatory filing of an ex vivo gene-editing therapy. Its CTX-001 for sickle cell anemia and beta-thalassemia has been co-developed with Vertex, could be submitted before the end of 2022.