CRISPR Patents Decision Favors Harvard and MIT’s Broad Institute
The decision is the latest action in a line of patent interferences over the revolutionary gene-editing technology.
The US Patent and Trademark Office (USPTO) Patent Trial Appeal Board (PTAB) has ruled that the Broad Institute of Harvard University and the Massachusetts Institute of Technology were the first to invent CRISPR/Cas9 editing in eukaryotic cells – not the group known collectively as CVC, which includes the University of California (UC), University of Vienna, and Emmanuelle Charpentier, PhD.1
Expressing its disappointment, theCVC group said it is considering various options to challenge the decision, while the University of California “believes the PTAB made a number of errors.”2
While Charpentier and Jennifer Doudna, PhD, UC Berkeley, are recognized as being the first to conceive the idea of CRISPR editing and were awarded the Nobel Prize in Chemistry for the “development of a method for genome editing” in 2020 as a result, the PTAB’s decision cited the lack of work by the CVC group specifically in eukaryotic cells.
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The CVC has over 40 issued US patents uninvolved with this interference related to CRISPR/Cas9 guiding in environments including eukaryotic cells. The Broad Institute has 13 CRISPR-related patents and 1 application that remain under challenge and the Institute is also involved in interferences with Toolgen and Sigma-Aldrich. The decision has reverberationsthrough multiple companies that are developing therapies with CRISPR/Cas9 technologies and lease the use of these technologies. Editas Medicines, who exclusively license the technology from Broad Institute, has multiple CRISPR therapies in early clinical studies for various indications.
Therapies in development by Editas include EDIT-101 for the potential treatment of Leber congenital amaurosis 10, currently being evaluated in a phase 1/2 study (NCT03872479); EDIT-301, which is currently being evaluated in a phase 1/2 study in sickle cell disease (SCD; NCT04853576) and whose investigational new drug application was approved in late 2021 for transfusion-dependent β thalassemia.
“While scientists in both groups made important scientific contributions to the field, this proceeding was to determine who invented the use of CRISPR/Cas9 for editing the DNA in eukaryotic cells, including human cells. We are pleased with the USPTO’s decision, ending the interference, and determining the Broad Institute’s innovative work to discover and use the CRISPR/Cas9 technology in human cells,” James C. Mullen, chairman, president, and chief executive officer, Editas Medicine, said in a statement.3 “The decision reaffirms the strength of our foundational intellectual property as we continue our work to develop life-changing medicines for people living with serious diseases.”
Meanwhile, the decision may affect current CRISPR-based therapies in development by CRISPR Therapeutics—for whom Charpentier serves as scientific cofounder—and Intellia Therapeutics, as both companies currently license CRISPR technology from the CVC group.
Intellia Therapeutics has in-vivo CRISPR candidates NTLA-2001 for transthyretin (ATTR) amyloidosis (NCT04601051) and NTLA-2002 for hereditary angioedema (NCT05120830) in early clinical studies as well as ex-vivo candidates OTQ923 / HIX763 for SCD (NCT04443907) and NTLA-5001 for acute myeloid leukemia (NCT05066165). CRISPR Therapeutics has a number of CRISPR candidates in clinical stages in indications such as β-thalassemia, SCD, hematologic malignancies, solid tumors, and diabetes.
Updated data recently released for NTLA-2001, which is being codeveloped by Intellia and Regeneron, showed deep and sustained reductions of transthyretinprotein in patients with hereditary ATTR amyloidosis with polyneuropathy. Investigators observed dose-dependent reductions in serum TTR, with peak reductions by day 28. Mean reductions were 52%, 87%, and 86% in 3 patients each in the first 3 cohorts and 93% in the 6 patients in the highest cohort.
In a statement, the Broad Institute reassured their commitment to keeping CRISPR technology open to the scientific community, stating that “Broad Institute has pressed for a joint licensing strategy, or patent pools, for more than eight years—before patents were issued to Broad or the University of California-Berkeley (UCB), with the hope of ensuring open, equitable, and streamlined access to these transformative tools. These efforts continue, and we remain optimistic.”4
