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US 10,238,753 B2Granted
Antisense conjugates for decreasing expression of DMPK
AssigneeValerion Therapeutics, LLC
InventorDustin D. Armstrong
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Abstract
The disclosure provides novel conjugates comprising antisense oligonucleotides that hybridize to a DMPK transcript and a 3E10 antibody or binding fragment thereof. Also considered are these conjugates further comprising MBNL1 polypeptides. Methods of treating myotonic dystrophy using these conjugates and kits comprising these conjugates are also considered. Wherein the conjugates are suitable for delivery to muscle cells.
Claims56
A conjugate comprising
- an antisense oligonucleotide that hybridizes to a DMPK transcript;
- and
- an antibody or antigen binding fragment, wherein the antibody or antigen binding fragment comprises a heavy chain variable domain comprising an amino acid sequence at least 95% identical to SEQ ID NO:
- 13, or wherein the antibody or antigen binding fragment comprises a light chain variable domain comprising an amino acid sequence at least 95% identical to SEQ ID NO:
- 14.
2 more elements
A conjugate comprising:
- an antisense oligonucleotide that hybridizes to a DMPK transcript;
- and
- an antibody or antigen binding fragment, wherein the antibody or antigen binding fragment comprises a heavy chain variable domain comprising the amino acid sequence of SEQ ID NO:
- 13 and a light chain variable domain comprising the amino acid sequence of SEQ ID NO:
- 14, or a humanized variant thereof.
2 more elements
A conjugate comprising
- an antisense oligonucleotide that hybridizes to a DMPK transcript;
- and
- an antibody or antigen binding fragment, wherein the antibody or antigen binding fragment comprises
- a VH CDR1 having the amino acid sequence of SEQ ID NO:
- 16;
- a VH CDR2 having the amino acid sequence of SEQ ID NO:
- 17;
- a VH CDR3 having the amino acid sequence of SEQ ID NO:
- 18;
- a VL CDR1 having the amino acid sequence of SEQ ID NO:
- 19;
- a VL CDR2 having the amino acid sequence of SEQ ID NO:
- 20;
- and
- a VL CDR3 having the amino acid sequence of SEQ ID NO:
- 21.
13 more elements
Dependencies are inferred from the claim text.