Muscle targeting complexes and uses thereof for treating myotonic dystrophy
AssigneeDyne Therapeutics, Inc.
InventorsRomesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, 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 expression or activity of a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Claims30
A composition comprising complexes, wherein each complex comprises an anti-transferrin receptor (TfR) antibody covalently linked to at least one oligonucleotides, wherein the antibody is a Fab and comprises a heavy chain comprising the amino acid sequence of SEQ ID NO:
- 101 and a light chain comprising the amino acid sequence of SEQ ID NO:
- 90, wherein each anti-TfR antibody of the complexes is on average covalently linked to 1 to 3 oligonucleotides, and wherein the oligonucleotide targets a Dystrophia Myotonica Protein Kinase (DMPK) RNA and comprises a region of complementarity to a sequence as set forth in any one of SEQ ID NO:
- 384-619, wherein the region of complementarity is 15-25 nucleotides in length.
Dependencies are inferred from the claim text.