Sarepta Therapeutics has been granted a patent for antisense oligomers that increase human type VII collagen expression, treating dystrophic epidermolysis bullosa. The compound targets exon 80 in the collagen mRNA, offering potential therapeutic benefits for related disorders. GlobalData’s report on Sarepta Therapeutics 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 Sarepta Therapeutics, siRNA gene silencing was a key innovation area identified from patents. Sarepta Therapeutics's grant share as of February 2024 was 39%. Grant share is based on the ratio of number of grants to total number of patents.

Antisense oligomers for treating dystrophic epidermolysis bullosa

Source: United States Patent and Trademark Office (USPTO). Credit: Sarepta Therapeutics Inc

A recently granted patent (Publication Number: US11911403B2) discloses an antisense oligomer compound with specific characteristics. The compound consists of 12 to 40 subunits, including a nucleotide analog with a modified internucleoside linkage or sugar moiety, along with a targeting sequence complementary to 12 or more contiguous nucleotides in a specific region of human type VII collagen pre-mRNA. This region includes the splice junction of exon 80/intron 80, with the targeting sequence chosen from specific SEQ ID numbers.

Furthermore, the patent details additional features of the antisense oligomer compound, such as the inclusion of a modified internucleoside linkage like phosphorothioate or phosphoramidate, a modified sugar moiety like peptide nucleic acid or locked nucleic acid subunits, and the potential conjugation of an arginine-rich cell-penetrating peptide. The compound's ability to hybridize to the target region effectively, as well as the specific nucleobases present in each subunit, are also outlined. The patent extends to cover a pharmaceutical composition containing the antisense oligomer compound and a variant of the compound that specifically hybridizes to the exon 80/intron 80 splice junction.

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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