公开(公告)号：WO2022240721A1

公开(公告)日：2022-11-17

申请号：PCT/US2022/028277

申请日：2022-05-09

Applicant: ENTRADA THERAPEUTICS, INC.

Inventor： QIAN, Ziqing ,  SETHURAMAN, Natarajan ,  KIERBAHDI, Mah ,  LIU, Haoming ,  SHEN, Xiulong ,  LI, Xiang

IPC: A61K47/64 ,  A61P37/02 ,  C07K7/64 ,  C12N15/113 ,  A61K47/645 ,  C12N2310/11 ,  C12N2310/3233 ,  C12N2310/3513 ,  C12N2320/33

Abstract: Compounds that include a cyclic cell penetrating peptide and a therapeutic moiety modulates Interferon Regulatory Factor – 5 (IRF-5) activity in an immune cell. The therapeutic moiety is a therapeutic protein, a small molecule, or an antisense compound. Methods include administering the compound to cells or subjects to modulate IRF-5 activity. Methods include treating diseases associated with IRF-5 using the compounds.

公开(公告)号：WO2022240758A1

公开(公告)日：2022-11-17

申请号：PCT/US2022/028354

申请日：2022-05-09

Applicant: ENTRADA THERAPEUTICS, INC.

Inventor： SHEN, Xiulong ,  LI, Xiang ,  QIAN, Ziqing ,  SETHURAMAN, Natarajan ,  LIU, Haoming

IPC: A61K31/7088 ,  A61K47/64 ,  C07K19/00 ,  C12N15/113 ,  C07K7/64 ,  A61K9/00 ,  A61K47/6455 ,  C12N2310/11 ,  C12N2310/3233 ,  C12N2310/341 ,  C12N2310/3513

Abstract: Compounds that include a cyclic cell penetrating peptide and a therapeutic moiety that modulates polyadenylation of a gene transcript. The therapeutic moiety may be an antisense compound (AC) that binds a target gene transcript. The AC may bind to at least a portion of a polyadenylation signal element (PSE) or may bind in sufficiently close proximity to the PSE to modulate polyadenylation of the target gene transcript. Methods include administering the aforementioned compounds to cells or subjects to modulate diseases or conditions.

公开(公告)号：WO2021127650A1

公开(公告)日：2021-06-24

申请号：PCT/US2020/066459

申请日：2020-12-21

Applicant: ENTRADA THERAPEUTICS, INC.

Inventor： QIAN, Ziqing ,  LI, Xiang ,  KHEIRABADI, Mah ,  DHANABAL, Mohanraj ,  LIU, Haoming ,  SHEN, Xiulong ,  KAMER, Kimberli ,  SETHURAMAN, Natarajan

IPC: A61K31/7088 ,  A61K38/00 ,  A61K47/42 ,  A61K47/64 ,  A61K48/00 ,  A61K49/00

Abstract: Provided herein are compounds comprising cyclic cell penetrating peptides and antisense compounds. Also provided herein are methods of modulating splicing, inhibiting or regulating translation, mediating degradation, blocking expansions of nucleotide repeats, and treating disease using the aforementioned compounds.

公开(公告)号：WO2022241408A1

公开(公告)日：2022-11-17

申请号：PCT/US2022/072217

申请日：2022-05-09

Applicant: ENTRADA THERAPEUTICS, INC.

Inventor： QIAN, Ziqing ,  DOUGHERTY, Patrick ,  KHEIRABADI, Mahboubeh ,  LIU, Haoming ,  SHEN, Xiulong

IPC: A61K47/64 ,  C07K7/64 ,  C12N15/113 ,  A61K38/00 ,  A61K47/6455 ,  A61P21/00 ,  A61P25/28 ,  C07K19/00 ,  C07K7/06 ,  C12N2310/11 ,  C12N2310/3233 ,  C12N2310/3513 ,  C12N2320/33

Abstract: Provided herein are compounds that include a cyclic cell penetrating peptide, a therapeutic moiety and a modulatory peptide, wherein the modulatory peptide modulates the tissue distribution and/or retention of the compound in vivo. Also provide herein are methods of modulating tissue distribution and/or retention of intracellular therapeutics using the aforementioned compounds.

公开(公告)号：WO2022240760A2

公开(公告)日：2022-11-17

申请号：PCT/US2022/028357

申请日：2022-05-09

Applicant: ENTRADA THERAPEUTICS, INC.

Inventor： QIAN, Ziqing ,  SETHURAMAN, Natarajan ,  SHEN, Xiulong ,  LIU, Haoming ,  LI, Xiang

IPC: A61K47/64 ,  C07K7/64 ,  C12N15/113 ,  A61K47/6455 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/3233 ,  C12N2310/3513 ,  C12N2320/33

Abstract: Compounds include at least one cyclic cell penetrating peptide (cCPP) conjugated to an antisense compound (AC). The AC modulates splicing of an RNA transcript. For example, the AC induces exon skipping. Exon skipping can result in downregulation of expression or activity of a protein. Exon skipping may cause a frameshift in a resulting mRNA. The frameshift may result in a premature termination codon. The frameshift may result in nonsense mediated decay.