公开(公告)号：WO2022043531A1

公开(公告)日：2022-03-03

申请号：PCT/EP2021/073803

申请日：2021-08-27

申请人： JANSSEN SCIENCES IRELAND UNLIMITED COMPANY ,  UNIVERSITE DE ROUEN NORMANDIE ,  CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE ,  INSTITUT NATIONAL DES SCIENCES APPLIQUEES DE ROUEN NORMANDIE

发明人： BEIGELMAN, Leonid ,  GUILLEMONT, Jérôme Émile Georges ,  PIETTRE, Serge ,  BRIOCHE, Julien ,  SUNCHU, Prabhakar

IPC分类号： C07H19/00 ,  C07H19/06 ,  C07H21/00 ,  C07D493/08 ,  A61K31/712 ,  A61P31/12

摘要： The present disclosure relates to ethylene-bridged nucleic acid (ENA) compounds, which may find use in therapy and/or prophylaxis. These ENA compounds may also find use as building blocks for preparing oligonucleotides, said oligonucleotides also finding use in therapy and/or prophylaxis. These compounds may be mono- or oligonucleotide derivatives comprising at least one of the following fragment: Formula (IV), wherein R1 is an optionally substituted C1-C6 alkyl moiety.

公开(公告)号：WO2021202907A2

公开(公告)日：2021-10-07

申请号：PCT/US2021/025427

申请日：2021-04-01

申请人： THE REGENTS OF THE UNIVERSITY OF MICHIGAN ,  SUN, Duxin ,  GAO, Wei ,  HU, Hongxiang ,  TRAORE, Mohamed Dit Mady ,  SONG, Yudong ,  WEN, Bo ,  HE, Miao ,  YUAN, Hebao ,  LI, Ruiting

发明人： SUN, Duxin ,  GAO, Wei ,  HU, Hongxiang ,  TRAORE, Mohamed Dit Mady ,  SONG, Yudong ,  WEN, Bo ,  HE, Miao ,  YUAN, Hebao ,  LI, Ruiting

IPC分类号： A61K31/015 ,  A61K31/135 ,  A61K31/165 ,  C07H19/06

摘要： The present invention relates to methods, compounds, and compositions for treating viral infections, including COVID-19 viral infections. In certain embodiments, the compositions comprise: i) a remdesivir analog, ii) remdesivir or a remdesivir analog, and a surfactant, a cyclodextrin, or a combination thereof, iii) nanoparticles comprising albumin and remdesivir or remdesivir analog, iv) liposomes comprising lipids and remdesivir or remdesivir analog; and/or v) microparticles comprising PLA and/or PLGA, and remdesivir or remdesivir analog. In certain embodiments, the compositions are aqueous (e.g., for intravenous administration). In other embodiments, the compositions are nebulized or in the form of a dry powder (e.g., for inhalation by an infected subject).

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

申请人： UNIVERSITY OF MASSACHUSETTS

发明人： KHVOROVA, Anastasia ,  ROUX, Loïc Maurice René Jean ,  YAMADA, Ken

IPC分类号： 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

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

公开(公告)号：WO2021167029A1

公开(公告)日：2021-08-26

申请号：PCT/JP2021/006222

申请日：2021-02-18

申请人： 国立大学法人大阪大学 ,  国立医薬品食品衛生研究所長が代表する日本国

发明人： 小比賀　聡 ,  山口　卓男 ,  小峯　飛々暉 ,  杉浦　隆也 ,  井上　貴雄 ,  吉田　徳幸

IPC分类号： C07H19/06 ,  C07H21/00 ,  C12N15/09

摘要： 架橋型ヌクレオシドおよびそれを用いたヌクレオチドを開示する。本発明のヌクレオシドは、以下の式（Ｉ）：　で表される。本発明の架橋型ヌクレオシドは、特定の臓器への集積などが懸念されるホスホロチオエート修飾核酸の代替とすることができ、工業的生産性にも優れている。

公开(公告)号：WO2021032075A1

公开(公告)日：2021-02-25

申请号：PCT/CN2020/109698

申请日：2020-08-18

申请人： SHANGHAI CHANGCHENGYIYAOKEJI COMPANY LIMITED

发明人： XU, Mingyan

IPC分类号： C07H19/06 ,  C07H19/11 ,  C07F9/6574 ,  A61K31/7068 ,  A61P35/00

摘要： Provided herein is a prodrug platform useful to deliver pharmaceutically active amines, amides and phenols and their use in the diagnosis, prevention and/or treatment of various diseases. Compared with the parent drug (e.g., Gemcitabine), the prodrugs show a significant overall safety improvement (therapeutic index (TI) improvement), especially in liver.

公开(公告)号：WO2020186124A1

公开(公告)日：2020-09-17

申请号：PCT/US2020/022519

申请日：2020-03-13

申请人： THE RESEARCH FOUNDATION FOR THE STATE UNIVERSITY OF NEW YORK

发明人： JU, Jingfang ,  FESLER, Andrew

IPC分类号： A61K45/06 ,  A61P35/00 ,  C07H21/02 ,  A61K48/00 ,  C07H19/06

摘要： The present disclosure provides modified microRNA nucleic acid compositions that have one or more cytosine and/or uracil bases replaced with gemcitabine or a 5-halouracil, respectively. More specifically, the present disclosure reveals that the replacement of cytosine nucleotides within a microRNA nucleotide sequence with a gemcitabine molecule increases the ability of the microRNA to inhibit cancer progression and tumorigenesis. In addition, the present disclosure reveals that the replacement of cytosine nucleotides within a microRNA nucleotide sequence with a gemcitabine molecule and replacement of uracil bases with 5-halouracil increases the ability of the microRNA to inhibit cancer development. As such, the present disclosure provides various modified nucleic acid (e.g., microRNA) compositions having gemcitabine molecules incorporated in their nucleic acid sequences and methods for using the same. The present disclosure further provides pharmaceutical compositions comprising the modified nucleic acid compositions, and methods for treating cancers using the same.

公开(公告)号：WO2020165334A1

公开(公告)日：2020-08-20

申请号：PCT/EP2020/053742

申请日：2020-02-13

申请人： DNA SCRIPT

发明人： SARAC, Ivo ,  BARBOUX, Cédric ,  NIYOMCHON, Supaporn ,  BEAUFILS, Florent ,  BUESCHLEB, Martin

IPC分类号： C07H1/00 ,  C07H19/04 ,  C07H19/06 ,  C07H19/16

摘要： The invention relates to a process for preparing a 3'-O-amino-ribonucleotide of formula (V) wherein (P) represents a phosphate group: (V). It also relates to a compound of formula (III), and its use as a precursor for the synthesis of a 3'-O-amino-ribonucleotide.

公开(公告)号：WO2020136170A2

公开(公告)日：2020-07-02

申请号：PCT/EP2019/086926

申请日：2019-12-23

申请人： ILLUMINA CAMBRIDGE LIMITED

发明人： FRANCAIS, Antoine ,  CRESSINA, Elena ,  CULLEY, Adam ,  MARIANI, Angelica ,  WU, Xiaolin ,  LIU, Xiaohai

IPC分类号： C07H19/06 ,  C07H19/10 ,  C07H19/16 ,  C07H19/20 ,  C07H21/00 ,  C12Q1/68

摘要： Embodiments of the present disclosure relate to nucleotide and nucleoside molecules with acetal or thiocarbamate 3ʹ-OH blocking groups. Also provided herein are methods to prepare such nucleotide and nucleoside molecules, and the uses of fully functionalized nucleotides containing the 3ʹ-OH blocking group for sequencing applications.

公开(公告)号：WO2019053659A1

公开(公告)日：2019-03-21

申请号：PCT/IB2018/057077

申请日：2018-09-14

申请人： ALIOS BIOPHARMA, INC.

发明人： GRYAZNOV, Sergei ,  HONG, Jin

IPC分类号： C07H1/00 ,  C07H19/06 ,  C07H19/067 ,  C07H19/16 ,  C07H19/167 ,  C07H19/24 ,  C07H21/00

摘要： The present disclosure relates to compounds and compositions containing 5'-phosphoramidite nucleoside monomers of formulae (I) and (II), and methods of making and use, wherein the substituents are as defined in the appended claims.

公开(公告)号：WO2019002237A1

公开(公告)日：2019-01-03

申请号：PCT/EP2018/067015

申请日：2018-06-26

申请人： ROCHE INNOVATION CENTER COPENHAGEN A/S

发明人： FUNDER, Erik Daa ,  HANSEN, Dennis Jul

IPC分类号： C07H1/00 ,  C07H1/02 ,  C07H19/06 ,  C07H19/16 ,  C07H21/00

摘要： The present invention relates to the field of stereodefined phosphorothioate oligonucleotides and to stereodefining nucleoside monomers and methods of synthesis of stereodefined oligonucleotides using said monomer. Herein are disclosed oligonucleotide enhanced synthesis methods where within a single elongation cycle there are repeated coupling and oxidation steps. The method results in an enhanced yield and higher purity of stereodefined phosphorothioate oligonucleotides