Precision Biosciences has been granted a patent for engineered meganucleases that can bind and cleave recognition sequences within a dystrophin gene. These meganucleases can be used to genetically modify cells and potentially treat Duchenne Muscular Dystrophy. The patent also covers pharmaceutical compositions containing these meganucleases or their encoding polynucleotides. 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.
Patent granted for engineered meganucleases for modifying dystrophin gene
A recently granted patent (Publication Number: US11753630B2) discloses a polynucleotide that includes two engineered meganucleases. The first engineered meganuclease binds and cleaves a specific nucleic acid sequence in the dystrophin gene, while the second engineered meganuclease binds and cleaves a different nucleic acid sequence in the same gene. The recognition sequences for these nucleases are provided in the patent as SEQ ID NO: 6 and SEQ ID NO: 10, respectively. The amino acid sequences for the first and second engineered meganucleases are also provided as SEQ ID NO: 43 and SEQ ID NO: 51, respectively.
The patent also describes a recombinant DNA construct that includes the aforementioned polynucleotide. This construct can be used for various applications in genetic engineering and gene therapy. The first and second nucleic acid sequences in the construct can be separated by an internal ribosome entry site (IRES) sequence or a nucleic acid sequence encoding a 2A peptide.
Furthermore, the patent discloses a recombinant adeno-associated virus (AAV) that contains the polynucleotide described earlier. Similar to the DNA construct, the first and second nucleic acid sequences in the AAV can be separated by an IRES sequence or a nucleic acid sequence encoding a 2A peptide. Additionally, the polynucleotide in the AAV can be driven by a muscle cell-specific promoter.
The patent also mentions that the recombinant AAV can have a capsid with the amino acid sequence of either SEQ ID NO: 182 or SEQ ID NO: 183. These capsid sequences are important for the viral vector's ability to deliver the engineered meganucleases to target cells.
In summary, the granted patent describes a polynucleotide containing two engineered meganucleases that can bind and cleave specific nucleic acid sequences in the dystrophin gene. The patent also covers recombinant DNA constructs and recombinant adeno-associated viruses that include this polynucleotide. These findings have potential applications in genetic engineering and gene therapy, particularly for targeting muscle cells.
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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
