Precision BioSciences granted NOA from USPTO for PCSK9 ARCUS Nuclease

Precision BioSciences announced it has received a Notice of Allowance from the U.S. Patent and Trademark Office USPTO for U.S. Patent Application No. 16/606,856, titled "Engineered meganucleases specific for recognition sequences in the PCSK9 gene." Corresponding international applications in this family have also been allowed in Europe, Australia, Mexico, Japan, and Israel since October 2022. Once issued, patents arising from these international applications will have standard expiration dates in October 2038, and the U.S. patent arising from the ‘856 application will have a standard expiration date in August 2040. The allowed composition of matter claims in these U.S. and international applications encompass a PCSK9-specific ARCUS nuclease. This ARCUS nuclease has been used preclinically for both gene deletion approaches for cardiovascular diseases such as familial hypercholesterolemia and, as importantly, a viable and safe site to insert a functional copy of a specific gene to restore function addressing other diseases requiring gene addition. "Due to their unique properties and Precision’s state-of-the-art engineering process, ARCUS nucleases are exceptionally well-suited for many hard-to-treat genetic conditions requiring gene insertion or addition and large gene excisions. These types of gene edits may be too complex for other gene editing technologies at the editing efficiencies needed for therapeutic benefit," said Jeff Smith, Co-Founder and Chief Research Officer at Precision BioSciences. "These patents further strengthen our intellectual property portfolio on our PCSK9 ARCUS nucleases which provide us with the potential capability and optionality to target the well-characterized PCSK9 gene for gene deletion through partnerships in cardiovascular diseases, such as familial hypercholesterolemia, as well as provide us the potential ability to more rapidly pioneer therapeutic gene insertion strategies for Precision’s own organic pipeline." In 2023, Precision expects to host an in vivo gene editing R&D event and present new preclinical data in Duchenne muscular dystrophy and hepatitis B. In addition, iECURE is targeting to file a CTA and/or IND for a product incorporating the Precision PCSK9 nuclease for ornithine transcarbamylase deficiency in the second half of 2023.