Intellia Therapeutics’ Statement on Recent U.S. Patent and Trademark Office Decision Relating to CRISPR/Cas9 Genome Editing Technology in Eukaryotic Cells

March 4, 2022 at 10:00 AM EST

Background

  • In May 2012, the Regents of University of California, University of Vienna, and Emmanuelle Charpentier (collectively, “CVC”) filed a patent application for their CRISPR/Cas9 genome editing technology. The CVC team led by Drs. Jennifer Doudna and Emmanuelle Charpentier promptly brought these discoveries to the public, and in June 2012 published a seminal paper in Science describing their breakthrough technology. That paper unleashed a flurry of scientific activity that has grown into the field of CRISPR/Cas9 genome editing.
  • In 2020, Dr. Doudna from the University of California, Berkeley and Dr. Charpentier, director at the Max Planck Institute for Infection Biology in Berlin, Germany, were awarded the Nobel Prize in Chemistry “for the development of a method for genome editing."
  • A few months after CVC’s initial patent filing, in December 2012, another academic group at the Broad Institute, Harvard University and the Massachusetts Institute of Technology (collectively, “Broad”) filed a patent application pertaining to its work with CRISPR/Cas9 genome editing in eukaryotic cells.
  • Since 2016, CVC and Broad have been involved in patent interference proceedings at the U.S. Patent and Trademark Office over several patents and patent applications pertaining to use of the CRISPR/Cas9 genome editing system in eukaryotic cells, that is, plants and animals.


Intellia’s Rights to CVC’s Intellectual Property

  • Intellia has an exclusive license to use CVC’s CRISPR/Cas9 IP estate for the development and commercialization of human therapeutics, with the exception of antibacterial and antifungal applications.
  • Additionally, Intellia has filed numerous patent applications covering its own CRISPR/Cas9 technological innovations, delivery applications and product candidates.
  • None of Intellia’s own patent applications are involved or impacted by the interference between the CVC and Broad, which only pertain to a subset of the in-licensed CVC CRISPR/Cas9 IP estate.


Recent Ruling by the U.S. Patent and Trademark Office’s (“USPTO”) Patent and Trial Appeal Board (“PTAB”)

  • On Feb. 28, 2022, the USPTO’s PTAB issued its decision that Broad scientists were the first to invent the use of CRISPR/Cas9 genome editing in eukaryotic cells.
  • This ruling affected 14 patent applications owned by CVC, which are now refused by the USPTO.


Patents Not Included in the Latest Interference Ruling

  • CVC has over 45 issued U.S. patents that were not involved in this interference. These patents cover CRISPR/Cas9 genome editing systems in all environments, including eukaryotic cells. More specifically, this includes for human therapeutics.
  • CVC also has issued patents to its foundational CRISPR/Cas9 systems in over 30 countries, including in the U.K., European Union, China, Japan, and others, that are not affected by this or any U.S. interference proceeding.


Intellia’s View on the Latest Interference Ruling

  • Intellia disagrees with, and is disappointed by the PTAB’s decision.
  • Intellia does not expect the PTAB’s ruling to impact its ability to discover, develop, or commercialize future CRISPR-based medicines.
  • Intellia continues to focus on developing new CRISPR/Cas9-based therapeutics and generating additional IP protection covering its many gene editing and product innovations.


Ongoing CRISPR IP Dispute Possibilities

  • CVC can appeal the PTAB’s decision.
  • Anticipated PTAB decisions in separate U.S. interferences between the Broad and two other companies, Toolgen and Sigma-Aldrich, may impact Broad’s granted patents.
  • The PTAB, in this case, did not rule on the validity or enforceability of the Broad patents involved in the interference. The Broad’s patents may still be challenged in later proceedings.
  • Parties may reach a resolution outside of legal proceedings.


At Intellia, we are confident that CVC will find a path forward and CVC remains in a strong IP position in the U.S. and worldwide. We ultimately believe that a future solution should include the best interest of patients who might benefit from novel CRISPR-based medicines.

Forward-Looking Statements

This statement contains “forward-looking statements” of Intellia within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding the intellectual property position and strategy of Intellia, its licensors or other parties from which it derives rights, including with respect to intellectual property regarding the CRISPR/Cas9 genome editing technology, or that of unrelated third parties; Intellia’s ability to develop and commercialize CRISPR/Cas9-based therapeutic products to address severe and life-threatening diseases; Intellia’s expectations around the potential implications of the patent interference and the Feb. 28, 2022, decision of the United States Patent and Trademark Office’s Patent Trial and Appeal Board (“PTAB”); Intellia’s scientific, business and financial plans and prospects; Intellia’s expectations on future proceedings of the PTAB’s February 28 decision and Intellia’s expectations on the IP position of Dr. Emmanuelle Charpentier, The University of California and the University of Vienna. Any forward-looking statements in this statement are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, risks related to Intellia’s ability to protect and maintain its position and rights regarding its intellectual property portfolio, risks related to the ability of Intellia’s licensors and other parties from which it derives rights to protect and maintain their intellectual property position and rights, risks related to the uncertainty of litigation and decisions by courts and other adjudicators, the risk that third parties own or control intellectual property necessary for Intellia to develop or commercialize its product candidates, and the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s subsequent filings with the Securities and Exchange Commission. All information in this statement is as of the date of the release, and Intellia Therapeutics undertakes no duty to update this information unless required by law.

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