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.

公开(公告)号：WO2022232411A2

公开(公告)日：2022-11-03

申请号：PCT/US2022/026760

申请日：2022-04-28

Applicant: EISAI R&D MANGEMENT CO., LTD.

Inventor： VATHIPADIEKAL, Vinod ,  MITASEV, Branko ,  EASLEY-NEAL, Courtney ,  CHOI, Hyeong Wook ,  FANG, Frank ,  WANG, John ,  VEMULA, Praveen ,  LEE, Jung Hwa

IPC: C12N15/113 ,  A61K31/712 ,  A61K31/7125 ,  A61P25/28 ,  C12N15/1138 ,  C12N2310/11 ,  C12N2310/314 ,  C12N2310/321 ,  C12N2310/3233 ,  C12N2320/33

Abstract: Novel antisense oligonucleotides that induce Exon-2 skipping in the CD33 gene during pre-mRNA splicing, and their use in the treatment of a neurodegenerative disease, such as Alzheimer's disease, are disclosed.

公开(公告)号：WO2022219409A2

公开(公告)日：2022-10-20

申请号：PCT/IB2022/000218

申请日：2022-04-15

Applicant: SIXFOLD BIOSCIENCE LTD.

Inventor： FOOT, George, William ,  ROSELL, Anna, Perdrix ,  RUSHWORTH, James, Luke

IPC: C12N15/11 ,  A61K47/69 ,  B82Y5/00 ,  C12N15/111 ,  C12N2310/314 ,  C12N2310/351 ,  C12N2310/3513 ,  C12N2310/3519 ,  C12N2320/32 ,  C12N2320/50 ,  C12N2330/30

Abstract: The invention provides therapeutic compositions that include nucleic acid nanoparticles for delivery of cargo and methods of using the same.

公开(公告)号：WO2021234459A2

公开(公告)日：2021-11-25

申请号：PCT/IB2021/000351

申请日：2021-05-24

Applicant: WAVE LIFE SCIENCES LTD.

Inventor： VARGEESE, Chandra ,  IWAMOTO, Naoki ,  APPONI, Luciano, H. ,  BUTLER, David, Charles Donnell ,  KANDASAMY, Pachamuthu ,  MARAPPAN, Subramanian ,  TRIPATHI, Snehlata ,  LIU, Wei ,  BEDEKAR, Mugdha ,  VATHIPADIEKAL, Vinod

IPC: C12N15/113 ,  C12N15/11 ,  C07H21/00 ,  C07H21/02 ,  C07H21/04 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/3233 ,  C12N2310/343 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2320/32

Abstract: The present disclosure provides double stranded oligonucleotides, compositions, and methods relating thereto. The present disclosure encompasses the recognition that structural elements of double stranded oligonucleotides, such as base sequence, chemical modifications (e.g, modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, and/or stereochemistry (e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)), and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g, RNA interference (RNAi) activity, stability, delivery, etc. The present disclosure also provides methods for treatment of diseases using provided double stranded oligonucleotide compositions, for example, in RNA interference.

公开(公告)号：WO2021195533A2

公开(公告)日：2021-09-30

申请号：PCT/US2021/024425

申请日：2021-03-26

Applicant: UNIVERSITY OF MASSACHUSETTS

Inventor： KHVOROVA, Anastasia ,  ROUX, Loïc Maurice René Jean ,  YAMADA, Ken

IPC: A61K48/00 ,  C07H19/06 ,  C07H19/16 ,  C07H19/067 ,  C07H21/02 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/31 ,  C12N2310/312 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3515 ,  C12N2320/51

Abstract: This disclosure relates to the synthesis of novel modified oligonucleotides. The synthesis of novel phosphoramidites are also provided.