公开(公告)号：WO2023034853A2

公开(公告)日：2023-03-09

申请号：PCT/US2022/075745

申请日：2022-08-31

Applicant: CITY OF HOPE

Inventor： KORTYLEWSKI, Marcin Tomasz ,  YU, Chunsong ,  SWIDERSKI, Piotr Marek

IPC: A61K31/522 ,  A61K31/7076 ,  A61K31/713 ,  A61K39/395 ,  A61K47/54 ,  A61P35/00 ,  A61K31/712 ,  C12N15/117 ,  C12N2310/11 ,  C12N2310/17 ,  C12N2310/315 ,  C12N2310/336 ,  C12N2310/344 ,  C12N2320/31

Abstract: The disclosure provides, inter alia, oligonucleotides comprising 6-thio-2'-deoxy-guanosine residues. The oligonucleotides comprising said residues are used in pharmaceutical compositions and methods for treating cancer. The invention also includes CpG oligodeoxynucleotides in combination with the 6-thio-2'-deoxy-guanosine residues in said pharmaceutical compositions and methods for treating cancer. Oligonucleotides of the invention may also include additional therapeutic moieties or exonuclease resistant moieties. The compositions and methods of the invention may employ additional pharmaceutical entities for the treatment of cancer.

公开(公告)号：WO2022029209A1

公开(公告)日：2022-02-10

申请号：PCT/EP2021/071824

申请日：2021-08-05

Applicant: F. HOFFMANN-LA ROCHE AG ,  HOFFMANN-LA ROCHE INC. ,  DICERNA PHARMACEUTICALS, INC.

Inventor： OTTOSEN, Søren ,  MUELLER, Henrik ,  ACHNECK, Hardean ,  FAMBROUGH, Douglas M. ,  BROWN, Bob Dale

IPC: C12N15/113 ,  A61K31/713 ,  A61P31/20 ,  C12N15/1131 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/531 ,  C12N2320/31 ,  C12N2320/35

Abstract: The present invention provides oligonucleotides for use in the treatment of hepatitis B or hepatitis B virus infection in a human patient.

公开(公告)号：WO2021096763A1

公开(公告)日：2021-05-20

申请号：PCT/US2020/059265

申请日：2020-11-06

Applicant: ALNYLAM PHARMACEUTICALS, INC.

Inventor： FOSTER, Donald ,  AGARWAL, Sagar ,  HUANG, Stephen, Albert ,  KIM, Jae

IPC: C12N15/113 ,  A61K31/713 ,  C07H21/02 ,  A61P9/12 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/351 ,  C12N2320/31 ,  C12N2320/35 ,  C12N2320/52

Abstract: The present invention relates to methods of inhibiting the expression of an AGT gene in a subject, as well as methods for treating subjects having an AGT-associated disorder, e.g., hypertension, using RNAi agents, e.g., double stranded RNAi agents, targeting the AGT gene. The invention also relates to methods of decreasing blood pressure levels in a subject using such RNAi agents to inhibit expression of an AGT gene.

公开(公告)号：WO2023039444A2

公开(公告)日：2023-03-16

申请号：PCT/US2022/076068

申请日：2022-09-07

Applicant: VERTEX PHARMACEUTICALS INCORPORATED

Inventor： FULGA, Tudor ,  XIN, Yurong ,  MIN, Yi-Li ,  LIU, Jianming ,  BOLUKBASI, Fatih

IPC: C12N15/113 ,  C12N9/22 ,  C12N15/10 ,  C12N15/86 ,  C12N15/907 ,  C12N2310/20 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2330/51 ,  C12N2750/14143

Abstract: Compositions and methods for treating Duchenne Muscular Dystrophy (DMD) and excising small portions of exon 51 of the DMD gene are encompassed.

公开(公告)号：WO2022271955A1

公开(公告)日：2022-12-29

申请号：PCT/US2022/034736

申请日：2022-06-23

Applicant: MUSC FOUNDATION FOR RESEARCH DEVELOPMENT

Inventor： MISRA, Suniti ,  GHATAK, Shibnath

IPC: C12N15/113 ,  A61K31/713 ,  A61K9/51 ,  A61P35/00 ,  A61K9/0019 ,  A61K9/1272 ,  A61K9/5138 ,  A61K9/5146 ,  C12N15/1138 ,  C12N2320/31 ,  C12N2320/32 ,  C12N2320/34

Abstract: The present invention generally relates to compositions comprising one or more modulator of one or more chemoresistance promoting molecule, one or more chemosensitivity promoting molecule, or combination thereof, and methods of use thereof. The present invention also relates to CD44v6 inhibitor compositions and methods of use for treating or preventing one or more disease or disorder, such as cancer.

公开(公告)号：WO2022147249A1

公开(公告)日：2022-07-07

申请号：PCT/US2021/065682

申请日：2021-12-30

Applicant: MAYO FOUNDATION FOR MEDICAL EDUCATION AND RESEARCH

Inventor： ACKERMAN, Michael J. ,  DOTZLER, Steven M. ,  GENDRON, William ,  BAINS, Sahej ,  KIM, Chang Sung ,  TESTER, David J.

IPC: A61K31/7088 ,  A61K31/713 ,  A61K38/17 ,  A61K48/00 ,  A61P9/00 ,  C07H21/02 ,  A01K2217/054 ,  A01K2227/107 ,  A01K2267/0375 ,  A61K31/7105 ,  A61K38/00 ,  A61K48/005 ,  C07K14/705 ,  C12N15/1138 ,  C12N15/86 ,  C12N2310/14 ,  C12N2320/31 ,  C12N2320/34 ,  C12N2740/16043 ,  C12N2750/14143

Abstract: Methods and materials for treating a mammal having a congenital disease (e.g., a congenital heart disease such as congenital long QT syndrome) are provided herein. For example, this document provides methods and materials for generating and using nucleic acids to treat a mammal having a congenital disease, where the nucleic acids can suppress expression of mutant disease-related alleles in the mammal while providing a replacement cDNA that does not contain the disease-related mutation(s).

公开(公告)号：WO2021231771A2

公开(公告)日：2021-11-18

申请号：PCT/US2021/032311

申请日：2021-05-13

Applicant: ARIZ PRECISION MEDICINE

Inventor： BOOKBINDER, Lonnie L. ,  VAISH, Narendra K. ,  NILES, Brad J. ,  FLYNN, Jonathan ,  NUNEZ, Nicole

IPC: C12N15/113 ,  A61K31/713 ,  C07K14/47 ,  C12N15/87 ,  C12N2310/14 ,  C12N2310/351 ,  C12N2310/3513 ,  C12N2320/31 ,  C12N2320/32 ,  C12Y201/01043

Abstract: Inhibitory RNA molecules that specifically inhibit mammalian RIZ2 expression, with therapeutic effect in cell proliferative diseases, such as cancer.

公开(公告)号：WO2020160453A1

公开(公告)日：2020-08-06

申请号：PCT/US2020/016182

申请日：2020-01-31

Applicant: IONIS PHARMACEUTICALS, INC.

Inventor： KIM, Youngsoo ,  LUO, Xiaolin ,  MACLEOD, Robert ,  FREIER, Susan, M. ,  BUI, Huynh-Hoa

IPC: A61K31/7088 ,  A61K31/712 ,  A61K31/7125 ,  A61P35/00 ,  C12N15/11 ,  C12N15/113 ,  A61K45/00 ,  A61K9/0012 ,  C12N15/1135 ,  C12N2310/315 ,  C12N2320/31

Abstract: The present embodiments provide methods, compounds, and compositions useful for inhibiting YAP1 expression, which may be useful for treating, preventing, or ameliorating a cancer associated with YAP1. Certain embodiments provided herein relate to methods of inhibiting YAP1 expression, which can be useful for treating, preventing, or ameliorating a cancer associated with YAP1 in an individual, by administration of a compound that targets YAP1. In certain embodiments, the compound can be a YAP specific inhibitor. In certain embodiments, the compound can be an antisense compound, oligomeric compound, or oligonucleotide targeted to YAP1.

公开(公告)号：WO2022097157A2

公开(公告)日：2022-05-12

申请号：PCT/IL2021/051331

申请日：2021-11-09

Applicant: 1E THERAPEUTICS, LTD.

Inventor： BACHELET, Ido ,  HOROWITZ, Almogit ,  ROSENBERG, Alexander ,  SHAPIRO, Anastasia ,  OSHRI, Ron ,  KOLODKIN-GAL, Ilana ,  LEVY-ZAMIR, Adva ,  KRIEGER, Gat ,  GILLIS, Ella ,  ITTAH, Shmulik

IPC: C12N15/113 ,  A61K31/711 ,  A61K31/7115 ,  A61K31/43 ,  A61K31/431 ,  A61P31/04 ,  C12N15/1137 ,  C12N2310/127 ,  C12N2310/315 ,  C12N2310/3341 ,  C12N2310/3513 ,  C12N2310/3515 ,  C12N2310/51 ,  C12N2320/31 ,  C12Y114/20 ,  C12Y203/02017 ,  C12Y305/02006

Abstract: An oligonucleotide is provided. The oligonucleotide comprising a nucleic acid sequence of at least one DNAzyme, the DNAzyme being capable of silencing at least one target gene of a bacteria to thereby render the bacteria susceptible to antibiotic treatment.

公开(公告)号：WO2022026387A1

公开(公告)日：2022-02-03

申请号：PCT/US2021/043182

申请日：2021-07-26

Applicant: ALIGOS THERAPEUTICS, INC.

Inventor： HONG, Jin ,  BEIGELMAN, Leonid ,  BHATTACHARYA, Aneerban ,  DE COSTA, N. Tilani S.

IPC: C12N15/113 ,  A61K31/712 ,  A61K31/7115 ,  A61P31/20 ,  A61K31/713 ,  A61K45/06 ,  C12N15/1131 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/351 ,  C12N2320/31

Abstract: Oligonucleotides that target hepatitis B virus (HBV) viral sequences, such as rcDNA, cccDNA, and HBV transcripts, are described herein. In addition, compositions and kits comprising such oligonucleotides are further described. Further disclosed herein are uses of such oligonucleotides and compositions to reduce rcDNA to cccDNA conversion, reduce cccDNA levels, and/or treat an HBV infection.