Muscle-targeting complexes and uses thereof
AssigneeDyne Therapeutics, Inc.
InventorsRomesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Jason P. Rhodes, Cody A. Desjardins, Brendan Quinn, John Najim
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Abstract
Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits activity of a disease allele associated with muscle disease. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Claims42
A complex comprising an anti-transferrin receptor (TfR) antibody covalently linked to a molecular payload configured to modulate expression or activity of a muscle disease gene, wherein the antibody comprises:
- a heavy chain variable region (VH) comprising the amino acid sequence of SEQ ID NO:
- 76;
- and a light chain variable region (VL) comprising the amino acid sequence of SEQ ID NO:
- 75.
A composition comprising complexes, wherein each complex comprises an anti-transferrin receptor (TfR) antibody covalently linked to at least one molecular payload configured to modulate expression or activity of a muscle disease gene, wherein the antibody comprises a heavy chain variable region (VH) comprising the amino acid sequence of SEQ ID NO:
- 76 and a light chain variable region (VL) comprising the amino acid sequence of SEQ ID NO:
- 75, wherein each anti-TfR antibody of the complexes is on average covalently linked to 1 to 3 molecular payloads.
Dependencies are inferred from the claim text.