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
Life of this patent
Estimating the term…
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.
Claims42
A complex comprising an anti-transferrin receptor (TfR) antibody covalently linked to an oligonucleotide,
- wherein the anti-TfR 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, and wherein the oligonucleotide targets a Dystrophia Myotonica Protein Kinase (DMPK) RNA.
A composition comprising complexes, wherein each complex comprises an anti-transferrin receptor (TfR) antibody covalently linked to at least one oligonucleotide, 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 oligonucleotides, and wherein the oligonucleotide targets a Dystrophia Myotonica Protein Kinase (DMPK) RNA.
Dependencies are inferred from the claim text.