Sarepta Therapeutics has been granted a patent for oligonucleotide analogues conjugated to carrier peptides. These compounds can be used for the treatment of various diseases by inhibiting protein expression or correcting aberrant mRNA splice products. The patent specifically claims a conjugate comprising a carrier peptide, a phosphorodiamidate morpholino oligomer (PMO), and a covalent attachment between the morpholino oligomer and the carrier peptide. 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 September 2023 was 39%. Grant share is based on the ratio of number of grants to total number of patents.
Conjugate of carrier peptide and oligonucleotide for disease treatment
A recently granted patent (Publication Number: US11732259B2) describes a conjugate that combines a carrier peptide, a phosphorodiamidate morpholino oligomer (PMO), and a covalent attachment between the two. The carrier peptide consists of amino acid subunits, with a glycine or proline amino acid at the carboxy terminus. The PMO has a substantially uncharged backbone and a targeting base sequence for binding to a specific nucleic acid target. The covalent attachment is formed through an amide bond to the carboxy-terminal glycine or proline, with an optional linker group.
The patent claims specify various aspects of the conjugate. The carrier peptide can have a glycine or proline amino acid at the carboxy terminus. It can consist of 4 to 40 amino acid subunits, or alternatively, 6 to 20 amino acid subunits. The carrier peptide can contain positively charged amino acids such as histidine, lysine, arginine, or combinations thereof. Arginine can be the sole positively charged amino acid or one of multiple positively charged amino acids. An arginine analog can also be used as a positively charged amino acid.
The claims further state that at least 20%, 50%, or 80% of the amino acid subunits in the carrier peptide are positively charged amino acids. In some embodiments, all amino acid subunits, except the carboxy terminal glycine or proline, are positively charged amino acids. The carrier peptide can have a specific sequence, such as (Rd)m, where Rd is independently arginine, histidine, or lysine, and m is an integer ranging from 2 to 12. Alternatively, each amino acid subunit, except the carboxy terminal glycine or proline, can be arginine.
Additionally, the carrier peptide can include hydrophobic amino acids with substituted or unsubstituted alkyl, alkenyl, alkynyl, aryl, or aralkyl side chains. The hydrophobic amino acid side chains can have at most one heteroatom for every six carbon atoms. The carrier peptide may contain two or more hydrophobic amino acids.
This patent provides a detailed description of a conjugate that combines a carrier peptide and a PMO for sequence-specific binding to a target nucleic acid. The various claims outline different configurations and compositions of the carrier peptide, including the presence of specific amino acids, the number of amino acid subunits, and the proportion of positively charged amino acids. The patent's findings could have implications for the development of targeted nucleic acid therapies.
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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
