公开(公告)号：WO2023081893A1

公开(公告)日：2023-05-11

申请号：PCT/US2022/079409

申请日：2022-11-07

Applicant: ENTRADA THERAPEUTICS, INC.

Inventor： QIAN, Ziqing ,  KHEIRABADI, Mahboubeh ,  STREETER, Matthew

IPC: A61K47/54 ,  A61K47/64 ,  A61P1/16

Abstract: Compounds are provided include a cell penetrating peptide, a therapeutic oligonucleotide, and a carbohydrate targeting moiety. The compounds may also include an exocyclic peptide. The compounds may be targeted to liver cells. The therapeutic oligonucleotide may be an oligonucleotide for treating a. disease or disorder associated with a liver cell.

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：WO2022213118A1

公开(公告)日：2022-10-06

申请号：PCT/US2022/071489

申请日：2022-03-31

Applicant: ENTRADA THERAPEUTICS, INC. ,  DOUGHERTY, Patrick ,  KHEIRABADI, Mahboubeh ,  LI, Xiang ,  QIAN, Ziqing

Inventor： DOUGHERTY, Patrick ,  KHEIRABADI, Mahboubeh ,  LI, Xiang ,  QIAN, Ziqing

IPC: C07K7/64 ,  C07K19/00 ,  C12N15/113 ,  A01K2217/075 ,  A01K2227/105 ,  A61K47/6455 ,  C12N15/111 ,  C12N15/87 ,  C12N15/907 ,  C12N2310/11 ,  C12N2310/3145 ,  C12N2310/3233 ,  C12N2310/3513 ,  C12N2320/32

Abstract: The present disclosure is directed to cell penetrating peptides, including cyclic cell penetrating peptides with high cytosolic delivery efficiency and reduced toxicity that are able to effectively deliver cargo inside a cell to treat a variety of conditions and diseases.

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：WO2019165183A1

公开(公告)日：2019-08-29

申请号：PCT/US2019/019117

申请日：2019-02-22

Applicant: ENTRADA THERAPEUTICS, INC. ,  SETHURAMAN, Natarajan ,  RUTH, Jason ,  TARTAGLIA, Lou ,  PEI, Dehua ,  QIAN, Ziqing

Inventor： SETHURAMAN, Natarajan ,  RUTH, Jason ,  TARTAGLIA, Lou ,  PEI, Dehua ,  QIAN, Ziqing

IPC: A61K35/28 ,  A61K48/00 ,  C12N5/078

Abstract: Disclosed herein are compositions and methods of treating disclosure provides for compounds for use in treating Mitochondrial Neurogastrointestinal Encephalopathy Syndrome (MNGIE). In some embodiments, the compounds have cell penetrating activity and thymidine phosphorylase activity. In certain embodiments, the compounds disclosed herein comprise: a) at least one cell-penetrating peptide (CPP) moiety; and b) a thymidine phosphorylase, or an active fragment or analog thereof (TP), wherein the CPP is coupled, directly or indirectly, to TP.

公开(公告)号：WO2023034818A1

公开(公告)日：2023-03-09

申请号：PCT/US2022/075693

申请日：2022-08-30

Applicant: ENTRADA THERAPEUTICS, INC.

Inventor： LI, Xiang ,  QIAN, Ziqing ,  KHEIRABADI, Mahboubeh ,  ZHOU, Ming ,  GIRGENRATH, Mahsweta ,  ESTRELLA, Nelsa ,  PEDDIGARI, Suresh ,  PATHAK, Anushree ,  SETHURAMAN, Natarajan ,  LIAN, Wenlong ,  LIU, Nanjun

IPC: A61K47/64 ,  C07K7/64 ,  C12N15/113 ,  C12N15/11

Abstract: Described herein in various embodiments are compositions comprising (a) a cell penetrating peptide (CPP) sequence; and (b) an antisense compound, wherein the antisense compound targets exon 45 of the DMD gene.

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.

公开(公告)号：WO2023034817A1

公开(公告)日：2023-03-09

申请号：PCT/US2022/075691

申请日：2022-08-30

Applicant: ENTRADA THERAPEUTICS, INC.

Inventor： LI, Xiang ,  QIAN, Ziqing ,  KHEIRABADI, Mahboubeh ,  WYSK, Mark ,  SETHURAMAN, Natarajan ,  LIAN, Wenlong ,  GIRGENRATH, Mahsweta ,  ESTRELLA, Nelsa

IPC: A61K47/64 ,  C07K7/64 ,  C12N15/113 ,  A61K38/00 ,  A61K47/6455 ,  A61P21/00 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/3233 ,  C12N2310/3513 ,  C12N2320/32 ,  C12N2320/33

Abstract: Described herein in various embodiments are compositions comprising (a) a cyclic peptide; and (b) an antisense compound, wherein the antisense compound targets exon 44 of the DMD gene in a pre-mRNA sequence.

公开(公告)号：WO2022271818A1

公开(公告)日：2022-12-29

申请号：PCT/US2022/034517

申请日：2022-06-22

Applicant: ENTRADA THERAPEUTICS, INC.

Inventor： SHEN, Xiulong ,  QIAN, Ziqing ,  DOUGHERTY, Patrick ,  KHEIRABADI, Mahboubeh ,  LI, Xiang

IPC: A61K47/64 ,  C07K7/64 ,  C12N15/113 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/0306 ,  A61K31/712 ,  A61K38/00 ,  A61K47/6455 ,  A61P21/00 ,  A61P25/00 ,  C07K19/00 ,  C12N2310/11 ,  C12N2310/3233 ,  C12N2310/3513

Abstract: Compounds comprising a cyclic peptide, such as a cyclic cell penetrating peptide, and an antisense compound are provided. The antisense compound binds to a gene having an expanded CTG repeat or a gene transcript having an expanded CUG repeat. The compounds can be delivered to subjects to treat diseases associated with expanded CTG·CUG repeats, such as myotonic dystrophy type 1 (DM1), spinocerebellar ataxia-8 (SCA8), and Huntington disease like-2 (HDL2).