Boston Children’s Hospital has been granted a patent for antisense oligonucleotides (AONs) designed to treat genetic disorders caused by transposable element insertions. The AONs target intronic sequences within genes and utilize modified backbones to induce exon skipping, offering a potential therapeutic approach. GlobalData’s report on Boston Children’s Hospital 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 Boston Children's Hospital, was a key innovation area identified from patents. Boston Children's Hospital's grant share as of May 2024 was 30%. Grant share is based on the ratio of number of grants to total number of patents.
Patent granted for antisense oligonucleotides for treating genetic disorders
A recently granted patent (Publication Number: US11999953B2) discloses a novel approach involving antisense oligonucleotides with modified backbones to target intronic sequences within a genome. These oligonucleotides, containing specific modifications like 2' methoxy ethyl or 2' O-methyl, are designed to bind to splice acceptor sites influenced by transposable elements or splice enhancer sequences, thereby inducing exon skipping. The sequences of these oligonucleotides bear a high degree of similarity to specific nucleotide sequences, as outlined in the patent.
Furthermore, the patent claims encompass variations of the antisense oligonucleotides, including those with modified nucleobases and sets comprising multiple oligonucleotides with similar functionalities. The application of these antisense oligonucleotides is extended to pharmaceutical compositions, where they are formulated with pharmaceutically acceptable excipients. Additionally, the inclusion of 5-methyl cytosine and/or 5-methyl uracil in these oligonucleotides is highlighted as part of the claimed invention. This patent represents a significant advancement in the field of genetic manipulation and targeted therapy, offering a precise and tailored approach to modulating gene expression through exon skipping mechanisms.
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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
