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公开(公告)号:WO2023067126A1
公开(公告)日:2023-04-27
申请号:PCT/EP2022/079346
申请日:2022-10-21
发明人: MORENO HERRERO, Jorge , HAAS, Heinrich , ERBAR, Stephanie , GARCIA FERNANDEZ, Jose Manuel , BENITO HERNANDEZ, Juan Manuel , LOPEZ FERNANDEZ, Jose , SÁNCHEZ MELLADO, Práxedes
IPC分类号: C07H15/14 , C07H15/26 , C07H21/02 , C07H21/04 , C07H99/00 , C07H1/00 , A61K47/54 , A61P35/00
摘要: The present disclosure provides compositions comprising an oligosaccharide of formula (I), wherein said compositions are useful for delivery of certain agents, including, for example, nucleic acids.
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公开(公告)号:WO2023023550A1
公开(公告)日:2023-02-23
申请号:PCT/US2022/075073
申请日:2022-08-17
IPC分类号: C07H21/02 , C07H19/16 , C12N15/113 , A61K31/7088 , A61K31/712 , A61K31/7125
摘要: The present disclosure provides oligomeric compounds (including oligomeric compounds that are antisense agents or portions thereof) comprising a modified oligonucleotide having at least one modified internucleoside linkage. In some embodiments, the disclosure provides oligomeric compounds and modified oligonucleotides comprising a central region in which purine-containing central region nucleosides are adjacent to methanesulfonyl phosphoramidate internucleoside linkages.
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公开(公告)号:WO2023007019A1
公开(公告)日:2023-02-02
申请号:PCT/EP2022/071478
申请日:2022-07-29
申请人: CUREVAC SE
IPC分类号: C07H19/00 , C07H21/00 , C12Q1/6806 , C07H19/20 , C07H21/02
摘要: The present invention is inter alia concerned with (A) a compound of formula (I) as defined herein or a salt, stereoisomer, tautomer or deuterated version thereof, (B) a cap analog comprising a 5' terminal acyclonucleoside, wherein the acyclonucleoside comprises a linear unbranched structure or a linear single-branched structure instead of a ribose, wherein the 5' terminal acyclonucleoside is optionally deuterated (C) an RNA molecule comprising at least three nucleotides and comprising a 5' end of formula (III) as defined herein, wherein the 5' end is optionally deuterated (D) an RNA molecule comprising at least three nucleotides and comprising a 5' terminal acyclonucleoside, wherein the acyclonucleoside comprises a linear unbranched structure or a linear single-branched structure instead of a ribose, wherein the 5' terminal acyclonucleoside is optionally deuterated (E) an in vitro method for synthesizing an RNA molecule, (F) the RNA molecule obtained thereby, (G) compositions comprising the RNA molecule, (H) kits comprising the compound of formula (I) or the cap analog, (I) uses as well as (J) methods as outlined herein.
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公开(公告)号:WO2023277168A1
公开(公告)日:2023-01-05
申请号:PCT/JP2022/026411
申请日:2022-06-30
申请人: 協和キリン株式会社 , 国立大学法人東海国立大学機構
IPC分类号: C12N15/11 , A61K31/7088 , A61K31/7115 , A61K31/712 , A61K31/7125 , A61K48/00 , A61P43/00 , C07H21/02 , C07H21/04 , C12N5/10 , C12N15/67 , C12P21/02
摘要: 本発明は、開始コドンから終止コドンまでの翻訳領域、5'側非翻訳領域、及びポリA鎖を含み、前記ポリA鎖を構成するヌクレオチドの65%以上が糖修飾ヌクレオチドである、ポリヌクレオチドに関する。
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公开(公告)号:WO2022236296A1
公开(公告)日:2022-11-10
申请号:PCT/US2022/072128
申请日:2022-05-05
发明人: BURNETT, Lisa , COTTER, Sara , TALKOWSKI, Michael
IPC分类号: C07H21/02 , C12N15/11 , C12N15/113 , C12N15/74 , C12Q1/68
摘要: Disclosed herein are methods of treating Prader-Willi Syndrome with polynucleotides or RNA.
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6.
公开(公告)号:WO2022191725A1
公开(公告)日:2022-09-15
申请号:PCT/PL2022/050013
申请日:2022-03-10
IPC分类号: C07H21/02 , C07H1/00 , C09B23/04 , C09B11/28 , C09B15/00 , C07D495/04 , C07D311/82 , C07D311/90 , C07D403/14 , C09B11/08 , C09B11/245 , C09B23/06 , C09B23/083
摘要: The subject of the invention is RNA analogs of formula 1, their preparation and application, inter alia, in microscopic observations, study of the gene expression process and monitoring of enzyme activity. wherein R1 is an (oligo)nucleotide containing group, R2 is a nucleobase, R3 is a functional group.
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公开(公告)号:WO2022187061A1
公开(公告)日:2022-09-09
申请号:PCT/US2022/017589
申请日:2022-02-23
申请人: BUTZLOFF, Peter
发明人: BUTZLOFF, Peter
IPC分类号: C01B32/152 , A61K31/197 , A61K31/198 , A61K33/44 , A61P25/28 , C07H21/02
摘要: A nanoparticle composed of buckminsterfullerene bonded to glutamine, gamma amino butyric acid (GABA) and adenosine triphosphate is provided. This nanoparticle helps to regulate the energy metabolism, neural excitability, and signal transduction within the synapse and between the astrocytes and the neuronal networks of the human brain when these become dysfunctional. It is intended as a treatment for Alzheimer's disease, Lewy Body disease and other neuropathological diseases of the glutamine-glutamate-GABA cycle. Utility for remediating comorbid sexual dysfunction is promoted, as well as the antioxidant and protein oligomer disassembly properties. The adenosine triphosphate adduct provides a reversible inorganic phosphate energy storage and supply, the glutamine adduct provides a stable reservoir of this amino acid that is resistant to breakdown by reactive oxygen species, reactive nitrogen species, and astrocytes. The GABA and buckminsterfullerene functional groups are anti-oxidants to treat the most oxidatively stressed regions at neural structures.
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公开(公告)号:WO2022178411A1
公开(公告)日:2022-08-25
申请号:PCT/US2022/017283
申请日:2022-02-22
IPC分类号: A61K31/713 , C12N15/113 , C07H21/02 , C12N15/11 , C12Q1/68
摘要: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the FLCN gene, as well as methods of inhibiting expression of FLCN, and methods of treating subjects that would benefit from reduction in expression of FLCN, such as subjects having a FLCN-associated disease, disorder, or condition, using such dsRNA compositions.
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公开(公告)号:WO2022162153A1
公开(公告)日:2022-08-04
申请号:PCT/EP2022/052067
申请日:2022-01-28
申请人: E-THERAPEUTICS PLC
IPC分类号: C07H15/04 , C07H15/08 , C07H21/00 , C07H21/02 , C07H21/04 , A61K31/713 , A61K47/54 , C07D207/404
摘要: The present invention relates to novel conjugated oligonucleotide compounds, which are suitable for therapeutic use. Additionally, the present invention provides methods of making these compounds, as well as methods of using such compounds for the treatment of various diseases and conditions.
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公开(公告)号:WO2022109171A1
公开(公告)日:2022-05-27
申请号:PCT/US2021/059944
申请日:2021-11-18
申请人: MODERNATX, INC.
摘要: The present invention relates to compositions and methods for the purification of ribonucleic acids including a polyA tail. The compositions and methods include a polystyrene divinylbenzene particle, a linker, and a poly-deoxythymidine oligonucleotide.
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