Precision Biosciences. has filed a patent for engineered meganucleases that can recognize and cleave a specific sequence in human mitochondrial DNA. These meganucleases can be used to modify or edit the mitochondrial DNA in genetically-modified eukaryotic cells. The patent also describes a mitochondria-targeting engineered meganuclease (MTEM) that includes a mitochondrial transit peptide (MTP) and a specific amino acid sequence. GlobalData’s report on Precision Biosciences gives a 360-degree view of the company including its patenting strategy. Buy the report here.

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According to GlobalData’s company profile on Precision Biosciences, Gene splicing using nucleases was a key innovation area identified from patents. Precision Biosciences's grant share as of September 2023 was 29%. Grant share is based on the ratio of number of grants to total number of patents.

Mitochondria-targeting engineered meganuclease for modifying human mitochondrial dna

Source: United States Patent and Trademark Office (USPTO). Credit: Precision Biosciences Inc

A recently filed patent (Publication Number: US20230295590A1) describes a mitochondria-targeting engineered meganuclease (MTEM) that can bind to and cleave a specific recognition sequence in mitochondrial genomes of eukaryotic cells. The MTEM consists of an engineered meganuclease attached to a mitochondrial transit peptide (MTP), with the engineered meganuclease having a specific amino acid sequence. The MTP is attached at the N-terminus of the engineered meganuclease. Additionally, the MTEM can be attached to a nuclear export sequence (NES) at the C-terminus.

The patent also covers a polynucleotide that encodes the MTEM, with the MTEM having the ability to bind and cleave the recognition sequence in mitochondrial genomes. The polynucleotide includes the nucleic acid sequence encoding the MTEM, the engineered meganuclease attached to the MTP, and the specific amino acid sequence of the engineered meganuclease. The MTP is attached at the N-terminus of the engineered meganuclease, and the MTEM can be attached to an NES at the C-terminus.

Furthermore, the patent describes a recombinant adeno-associated virus (AAV) that includes the polynucleotide encoding the MTEM. The MTEM in the recombinant AAV can bind and cleave the recognition sequence in mitochondrial genomes. The polynucleotide includes a promoter that is operably linked to the nucleic acid sequence encoding the MTEM. The MTP is attached at the N-terminus of the engineered meganuclease, and the MTEM can be attached to an NES at the C-terminus. The recombinant AAV can have an AAV9 capsid.

In summary, the patent describes a mitochondria-targeting engineered meganuclease (MTEM) that can bind and cleave a specific recognition sequence in mitochondrial genomes. The MTEM can be attached to a mitochondrial transit peptide (MTP) and a nuclear export sequence (NES). The patent also covers a polynucleotide encoding the MTEM and a recombinant adeno-associated virus (AAV) containing the polynucleotide. The AAV can have an AAV9 capsid and be driven by a CAG promoter.

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData Patent Analytics tracks bibliographic data, legal events data, point in time patent ownerships, and backward and forward citations from global patenting offices. Textual analysis and official patent classifications are used to group patents into key thematic areas and link them to specific companies