基本信息:
- 专利标题： MUSCLE TARGETING COMPLEXES FOR TREATING DYSTROPHINOPATHIES
- 申请号：PCT/US2022/078975 申请日：2022-10-31
- 公开(公告)号：WO2023077120A1 公开(公告)日：2023-05-04
- 发明人： WEEDEN, Timothy , VIEIRA, Benjamin , DESJARDINS, Cody, A. , SUBRAMANIAN, Romesh, R. , QATANANI, Mohammed, T. , QUINN, Brendan , NAJIM, John , SHEN, Peiyi
- 申请人： DYNE THERAPEUTICS, INC.
- 申请人地址： 1560 Trapelo Road
- 专利权人： DYNE THERAPEUTICS, INC.
- 当前专利权人： DYNE THERAPEUTICS, INC.
- 当前专利权人地址： 1560 Trapelo Road
- 代理机构： DAWSON, Cara, B. et al.
- 优先权： US63/274,306 2021-11-01
- 主分类号： A61K47/56
- IPC分类号： A61K47/56 ; A61K39/395 ; A61K47/50 ; A61K47/54 ; A61K47/55 ; A61P21/00
摘要：
Aspects of the disclosure relate to compositions comprising a plurality of complexes comprising an antibody (e.g., anti-TfRl antibody) covalently linked to one or more oligonucleotides (e.g. a phosphorodiamidate morpholino oligomer), each oligonucleotide being covalently linked at a linkage site represented by a lysine (K) residue of the antibody. In some embodiments, the antibody comprises a heavy chain comprising a heavy chain variable region (VH) and a heavy chain constant region, and a light chain comprising a light chain variable region (VL) and a light chain constant region, wherein at least 80% (e.g., 80%-98%, 80%-95%, 80%-90%, 85%-98%, 85%-95%, 85%- 90%, 90%-98%, or 90%-95%) of the light chain constant regions of the antibodies of the complexes in the composition are independently covalently linked to an oligonucleotide at a linkage site represented by K188 (based on Kabat numbering) and/or a linkage site represented by K190 (based on Kabat numbering) of the light chain constant region of each antibody.
IPC结构图谱:
| A | 人类生活必需 |
| --A61 | 医学或兽医学；卫生学 |
| ----A61K | 医用、牙科用或梳妆用的配制品 |
| ------A61K47/00 | 以所用的非有效成分为特征的医用配制品，如载体、惰性添加剂 |
| --------A61K47/50 | .非有效成分被化学键合到有效成分上，例如聚合物－药物结合物 |
| ----------A61K47/51 | ..非有效成分为改性剂 |
| ------------A61K47/56 | ...该改性剂是有机大分子化合物，例如寡聚的、聚合的或树枝状的分子 |