Human CRISPR Trials Set to Get Underway, with Patents Pending
International patent and U.S. courts agencies are once again setting up to possibly decide the course of clinical research, genomics and personalized drug development, this time with CRISPR.
But these court and agency decisions could leave large and small companies alike subject to acquiring commercial licenses to use the patented technology in order to effectively research, develop and deploy new therapies, according to Mark Barnes, a partner at the international law firm Ropes & Gray, who specializes in issues surrounding clinical research.
Both legacy and start-up biotech companies could become dependent on the abilities of researchers to invent new CRISPR methods, obtain patents and license them, Barnes said.
“If I were their [general counsel] or their [intellectual property] counsel, I would be paying a lot of attention to — in all the countries around the world, and in all the different jurisdictions — what patents are being applied for, and how would they affect what my scientists are doing,” he said. In addition, the holder could possibly maintain a monopoly on the technology over the patent’s 20-year lifespan.
The European Patent Office ruled to revoke the main patent on CRISPR-Cas9, as filed by the Broad Institute of MIT and Harvard University. The European patent was found to have omitted the name of one of the inventors listed on previous applications, Luciano Marraffini of Rockefeller University.
The Broad Institute described the EPO’s denial of the patent as “based on a technical formality,” and promised to appeal the decision. Meanwhile, several competing companies and universities have filed for European patents on eukaryotic applications.
The decision diverges from previous patent wins, including a U.S. settlement reached just days before the EPO decision, between the Broad Institute and Rockefeller University, regarding the inventorship and ownership of patents covering the use of CRISPR-Cas9 in eukaryotic cells.
Rockefeller asserted that Marraffini — who served as co-author with Broad’s Feng Zhang on a 2013 paper published in Science describing the technology — should have been listed in patent filings. Following binding arbitration, it was decided the patents will remain unchanged. Separate patents on the use of CRISPR-Cas9 in prokaryotic cells list Marraffini and Zhang as co-inventors and Rockefeller and Broad as joint owners.
In a high-profile February 2017 decision, the U.S. Patent and Trademark Office’s Patent Trial and Appeal Board ruled that the Broad Institute’s work in eukaryotic cells did not infringe or interfere with an earlier CRISPR patent filed by UC Berkeley. That decision was appealed to the Federal Circuit, where oral arguments have yet to be scheduled.
Meanwhile, the NIH launched a new research initiative aimed at developing new gene-editing technologies for treating patients — citing widespread interest and investment in the technology, but still many remaining challenges that prevent broad adoption, the NIH said.
The $190 million, six-year program in somatic cell genome editing plans to develop new and improved editor systems and assays for testing safety and efficacy in human cells — eventually assembling a toolkit to be shared with the scientific community.
The announcement comes amid reports that Chinese universities have already begun using CRISPR technologies on dozens of human patients in clinical trials. ClinicalTrials.gov lists at least 10 CRISPR studies based in China. Meanwhile, the first U.S. CRISPR clinical trial is scheduled to begin by the end of the month, sponsored by the University of Pennsylvania.
The Phase I study, NCT03399448, plans to begin enrolling 18 adult patients diagnosed with relapsed-refractory multiple myeloma, melanoma, synovial sarcoma or myxoid/round cell liposarcoma. Patients will receive treatment with autologous T cells edited by CRISPR to target NY-ESO-1, a tumor antigen.