The U.S. Court of Appeals for the Federal Circuit has instructed the U.S. Patent and Trademark Office’s Patent Trial and Appeal Board (PTAB) to reassess its 2022 decision regarding the invention of CRISPR-Cas9 gene editing technology. The court found that the PTAB used an incorrect standard in determining that scientists at the Broad Institute were the first to invent CRISPR-Cas9 for use in plant, animal, and fungal cells.

CRISPR-Cas9 is a significant advancement in DNA manipulation, credited to Jennifer Doudna from the University of California, Berkeley, Emmanuelle Charpentier formerly at Umeå University in Sweden, and their colleagues. These researchers are collectively known as CVC (California-Vienna-Charpentier). They received the 2020 Nobel Prize in Chemistry for this breakthrough.

Jeff Lamken, representing CVC in appeal proceedings, stated: “Today’s decision creates an opportunity for the PTAB to reevaluate the evidence under the correct legal standard and confirm what the rest of the world has recognized: that the Doudna and Charpentier team were the first to develop this groundbreaking technology for the world to share.”

The appellate court noted that relevant evidence was not considered by PTAB when it ruled Broad conceived of CRISPR-Cas9 before CVC. The court vacated PTAB's determination on conception and remanded it for further proceedings with instructions to reconsider consistent with their opinion.

CVC initially published their work on CRISPR-Cas9 gene editing in June 2012 in Science journal. Their method involved using three components: Cas9 enzyme, crRNA, and tracrRNA. They later demonstrated a streamlined process using a single-guide RNA.

The Broad Institute filed its patent application later in December 2012 for eukaryotic cell applications of CRISPR-Cas9. At that time, patents were awarded based on who invented first rather than who filed first.

In 2022, PTAB ruled Broad was first to invent CRISPR-Cas9 usage in eukaryotic cells—a decision now contested by CVC through appeal due to alleged errors including misapplication of legal standards.

CVC contended that other labs achieved similar results shortly after their publication using standard lab practices; hence Broad did not innovate anything new beyond these established methods.

Worldwide research on CRISPR continues rapidly despite ongoing patent disputes. The FDA and UK regulators have approved treatments using this technology for sickle cell disease.

University of California promotes commercialization through licensing agreements while maintaining nonprofit access rights. Notably, a collaboration between CRISPR Therapeutics and Vertex Pharmaceuticals led to conditional approval last year for a therapy targeting sickle-cell disease.

Research collaborations continue aiming at improving therapies potentially reducing or eliminating pre-treatment procedures like bone marrow destruction prior reinfusion post-CRISPR correction.