Dyne Therapeutics has filed a patent for complexes that consist of a muscle-targeting agent linked to a molecular payload. The muscle-targeting agent binds to a specific receptor on muscle cells, while the molecular payload enhances the expression or activity of a functional dystrophin protein. The molecular payload can be an oligonucleotide, such as an antisense oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele. One claim of the patent involves a complex comprising an anti-transferrin receptor antibody covalently linked to a molecular payload that promotes the expression or activity of a DMD gene. The antibody’s heavy and light chain variable regions have specific amino acid sequences. GlobalData’s report on Dyne 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 Dyne Therapeutics, Antibody-conjugate nanoparticles was a key innovation area identified from patents. Dyne Therapeutics's grant share as of September 2023 was 8%. Grant share is based on the ratio of number of grants to total number of patents.
The patent is filed for a complex that promotes dmd gene expression
A recently filed patent (Publication Number: US20230285586A1) describes a complex that includes an anti-transferrin receptor (TfR) antibody covalently linked to a molecular payload. The complex is designed to promote the expression or activity of a DMD gene, which is associated with dystrophinopathy. The antibody in the complex has specific characteristics, including a heavy chain variable region (VH) and/or a light chain variable region (VL) that have amino acid sequences at least 95% identical to specific sequences mentioned in the patent.
The patent claims also specify that the antibody can be in various forms, such as a Fab fragment, a Fab' fragment, a F(ab')2 fragment, a scFv, a Fv, or a full-length IgG. In particular, the patent highlights the use of a Fab fragment of the antibody.
The molecular payload in the complex is an oligonucleotide, which is a short DNA or RNA molecule. This oligonucleotide is designed to promote exon skipping in a DMD RNA, specifically targeting exon 8 to exon 55. The patent further describes the oligonucleotide having regions of complementarity to exonic splicing enhancers (ESEs) of a DMD transcript, as well as specific target sequences mentioned in the patent.
The patent also mentions that the oligonucleotide can be 20-30 nucleotides in length and can have specific sequences listed in the patent. Additionally, the oligonucleotide can include phosphorodiamidate morpholinos, which are a type of modified nucleotide.
The complex of the anti-TfR antibody and the oligonucleotide can be used to promote the expression or activity of a DMD protein in cells, particularly muscle cells. This method can potentially be used to treat individuals with a mutated DMD allele associated with dystrophinopathy. The patent suggests administering the complex to the subject in an effective amount for therapeutic purposes.
In summary, the patent describes a complex comprising an anti-TfR antibody and a molecular payload, specifically an oligonucleotide, for promoting the expression or activity of a DMD gene. The antibody and oligonucleotide have specific characteristics and sequences mentioned in the patent. The complex can be used to promote the expression or activity of a DMD protein in cells, potentially offering a treatment option for individuals with dystrophinopathy.
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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.
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
