Precision Biosciences. has been granted a patent for engineered meganucleases that target and modify the dystrophin gene, potentially aiding in the treatment of Duchenne Muscular Dystrophy. The patent includes methods for creating genetically modified cells and pharmaceutical compositions containing these meganucleases. 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 June 2024 was 27%. Grant share is based on the ratio of number of grants to total number of patents.
Engineered meganucleases for modifying dystrophin gene
The granted patent US12006522B2 encompasses a series of claims related to engineered meganucleases designed to target and cleave specific nucleic acid sequences within the dystrophin gene. Central to the claims is a polynucleotide that encodes an engineered meganuclease, characterized by a specific recognition sequence (SEQ ID NO: 6) and an amino acid sequence (SEQ ID NO: 43). The claims further detail that this polynucleotide can be linked to a promoter, which may be specific to muscle cells, enhancing the targeted expression of the meganuclease in relevant tissues. Additionally, the patent includes provisions for the polynucleotide to exist as mRNA and as part of recombinant DNA constructs.
Moreover, the patent outlines the creation of recombinant viruses, specifically recombinant adeno-associated viruses (AAV), that incorporate the polynucleotide encoding the engineered meganuclease. These viruses can also feature capsid proteins with specified amino acid sequences (SEQ ID NO: 182 and SEQ ID NO: 183) and may include muscle cell-specific promoters to ensure targeted delivery and expression. The claims collectively emphasize the potential for these engineered meganucleases and their delivery systems to facilitate precise genetic modifications, particularly in muscle cells, which could have implications for therapeutic strategies in conditions related to the dystrophin gene.
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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
