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AssigneeDyne Therapeutics, Inc.
InventorsNelson Hsia, Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Brendan Quinn, Cody A. Desjardins, John Najim, Sean Spring
Estimating the term…
Aspects of the disclosure relate to oligonucleotides designed to target DUX4 RNAs and targeting complexes for delivering the oligonucleotides to cells (e.g., muscle cells) and uses thereof, particularly uses relating to treatment of disease (e.g., FSHD). Wherein a complex comprises an anti-transferrin receptor 1 (TfR1) antibody covalently linked to an oligonucleotide configured for reducing expression or activity of DUX4.
A complex comprising an anti-transferrin receptor 1 (TfR1) antibody covalently linked to an oligonucleotide configured for reducing expression or activity of DUX4, wherein the anti-TfR1 antibody comprises a heavy chain complementarity determining region 1 (CDR-H1), a heavy chain complementarity determining region 2 (CDR-H2), a heavy chain complementarity determining region 3 (CDR-H3), a light chain complementarity determining region 1 (CDR-L1), a light chain complementarity determining region 2 (CDR-L2), a light chain complementarity determining region 3 (CDR-L3) of any of the anti-TfR1 antibodies listed in Tables 2-7 and wherein the oligonucleotide comprises an antisense strand comprising a region of complementarity to a DUX4 sequence as set forth in SEQ ID NO:
An oligonucleotide comprising the nucleotide sequence of any one of SEQ ID NOs:
Dependencies are inferred from the claim text.