Ionis Pharmaceuticals has developed methods, compounds, and compositions to reduce DMPK mRNA and protein expression in animals, particularly targeting CUGexp DMPK RNA to treat myotonic dystrophy. A patented compound is a single-stranded modified oligonucleotide with specific nucleobase sequences. GlobalData’s report on Ionis Pharmaceuticals 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 Ionis Pharmaceuticals, Nucleic acid active pharmaceutical ingredient was a key innovation area identified from patents. Ionis Pharmaceuticals's grant share as of May 2024 was 34%. Grant share is based on the ratio of number of grants to total number of patents.
A recently granted patent (Publication Number: US11981897B2) discloses a compound comprising a single-stranded modified oligonucleotide with specific characteristics. The compound consists of 16-30 linked nucleosides with a nucleobase sequence that is 100% complementary to a specific portion of a defined sequence (nucleobases 10195 to 10294 of SEQ ID NO: 2). The modified oligonucleotide must be at least 90% complementary to SEQ ID NO: 2 over its entirety and includes at least one modified internucleoside linkage and/or a modified nucleoside with a modified sugar. Various embodiments of the compound involve different modifications, such as phosphorothioate internucleoside linkages, modified sugars like bicyclic sugars, and 2'-substituted nucleosides.
Furthermore, the patent claims cover compositions containing the disclosed compounds along with pharmaceutically acceptable carriers or diluents. Additionally, methods for treating type 1 myotonic dystrophy (DM1) in animals are described, involving the administration of the compounds as specified in the claims. These methods aim to address the therapeutic application of the compounds in managing DM1, showcasing the potential for these modified oligonucleotides to be utilized in treating genetic disorders effectively. The patent provides a detailed framework for the design and application of these compounds, highlighting their specificity and potential in therapeutic interventions for DM1.
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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
