MX2024001581A - Derivados de ribosa modificados con 1'-alquilo y metodos de uso. - Google Patents

Derivados de ribosa modificados con 1'-alquilo y metodos de uso.

Info

Publication number
MX2024001581A
MX2024001581A MX2024001581A MX2024001581A MX2024001581A MX 2024001581 A MX2024001581 A MX 2024001581A MX 2024001581 A MX2024001581 A MX 2024001581A MX 2024001581 A MX2024001581 A MX 2024001581A MX 2024001581 A MX2024001581 A MX 2024001581A
Authority
MX
Mexico
Prior art keywords
methods
alkyl modified
modified ribose
ribose derivatives
linker compounds
Prior art date
Application number
MX2024001581A
Other languages
English (en)
Spanish (es)
Inventor
Weimin Wang
Xiaochuan Cai
Original Assignee
Sanegene Bio Usa Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sanegene Bio Usa Inc filed Critical Sanegene Bio Usa Inc
Publication of MX2024001581A publication Critical patent/MX2024001581A/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D307/00—Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom
    • C07D307/02—Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom not condensed with other rings
    • C07D307/04—Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
    • C07D307/18—Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom not condensed with other rings having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • C07D307/20—Oxygen atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/655—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having oxygen atoms, with or without sulfur, selenium, or tellurium atoms, as the only ring hetero atoms
    • C07F9/65515—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom having oxygen atoms, with or without sulfur, selenium, or tellurium atoms, as the only ring hetero atoms the oxygen atom being part of a five-membered ring
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6558—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system
    • C07F9/65586—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing at least two different or differently substituted hetero rings neither condensed among themselves nor condensed with a common carbocyclic ring or ring system at least one of the hetero rings does not contain nitrogen as ring hetero atom
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H13/00—Compounds containing saccharide radicals esterified by carbonic acid or derivatives thereof, or by organic acids, e.g. phosphonic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/26—Acyclic or carbocyclic radicals, substituted by hetero rings
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MX2024001581A 2021-08-05 2022-08-05 Derivados de ribosa modificados con 1'-alquilo y metodos de uso. MX2024001581A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163229628P 2021-08-05 2021-08-05
PCT/US2022/039517 WO2023014938A1 (en) 2021-08-05 2022-08-05 1'-alkyl modified ribose derivatives and methods of use

Publications (1)

Publication Number Publication Date
MX2024001581A true MX2024001581A (es) 2024-04-18

Family

ID=83049985

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024001581A MX2024001581A (es) 2021-08-05 2022-08-05 Derivados de ribosa modificados con 1'-alquilo y metodos de uso.

Country Status (10)

Country Link
US (2) US12465647B2 (enExample)
EP (1) EP4380946A1 (enExample)
JP (2) JP7842850B2 (enExample)
KR (1) KR20240070519A (enExample)
CN (1) CN117980315A (enExample)
AU (1) AU2022324471A1 (enExample)
CA (1) CA3227661A1 (enExample)
IL (1) IL310547A (enExample)
MX (1) MX2024001581A (enExample)
WO (1) WO2023014938A1 (enExample)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230227480A1 (en) * 2021-10-05 2023-07-20 Sanegene Bio Usa Inc. Polyhydroxylated cyclopentane derivatives and methods of use
WO2024081954A2 (en) 2022-10-14 2024-04-18 Sanegene Bio Usa Inc. Small interfering rna targeting c3 and uses thereof
EP4705312A1 (en) * 2023-05-03 2026-03-11 Sanegene Bio USA Inc. Lipid-based conjugates for systemic, central nervous system, peripheral nervous system, and ocular delivery
AU2024301344A1 (en) * 2023-07-24 2026-01-29 Sanegene Bio Usa Inc. Lipid-based enhancement agent for rna delivery and therapy
CN116854754B (zh) 2023-09-01 2023-12-12 北京悦康科创医药科技股份有限公司 一种含有核糖环或其衍生结构的GalNAc化合物及其寡核苷酸缀合物
WO2025143262A1 (ja) 2023-12-28 2025-07-03 日産化学株式会社 カルバモイルエチル修飾を含有する二本鎖rna
TW202608456A (zh) * 2024-05-09 2026-03-01 大陸商石藥集團中奇製藥技術(石家莊)有限公司 GalNAc衍生物及其寡聚核苷酸綴合物
WO2025256544A1 (zh) * 2024-06-11 2025-12-18 福建盛迪医药有限公司 一种1-氨烷基修饰的呋喃核糖衍生物的制备方法
WO2025264616A1 (en) * 2024-06-18 2025-12-26 Sanegene Bio Usa Inc. Small interfering rna targeting cfb and uses thereof
WO2026017140A2 (en) 2024-07-18 2026-01-22 Sanegene Bio Usa Inc. Small interfering rna targeting fxi and uses thereof
WO2026030554A1 (en) * 2024-07-31 2026-02-05 Sanegene Bio Usa Inc. Saccharide-based conjugates for rna delivery and therapy
CN118638166B (zh) 2024-08-15 2024-12-17 北京悦康科创医药科技股份有限公司 含有三氮唑结构的GalNAc化合物及其寡核苷酸缀合物
WO2026041067A2 (en) 2024-08-20 2026-02-26 Sanegene Bio Usa Inc. Small interfering rna targeting myostatin (mstn) and uses thereof
WO2026046411A1 (en) 2024-09-02 2026-03-05 Sanegene Bio Usa Inc. Small interfering rna targeting plasminogen (plg) and uses thereof
WO2026092658A2 (en) 2024-10-31 2026-05-07 Sanegene Bio Usa Inc. Small interfering rna targeting amyloid beta precursor protein (app) and uses thereof
WO2026102144A2 (en) * 2024-11-06 2026-05-15 Sanegene Bio Usa Inc. Oligonucleotide conjugates of phosphocholine lipid derivatives
US20260226475A1 (en) 2025-01-27 2026-08-06 Sanegene Bio Usa Inc. Small interfering rna targeting acvr1c and uses thereof

Family Cites Families (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US154A (en) 1837-03-30 Improvement in machines for cutting the threads of wood-screws
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US5453566A (en) 1986-03-28 1995-09-26 Calgene, Inc. Antisense regulation of gene expression in plant/cells
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
GB8822492D0 (en) 1988-09-24 1988-10-26 Considine J Apparatus for removing tumours from hollow organs of body
CA2039718C (en) 1989-08-31 2003-02-25 John J. Rossi Chimeric dna-rna catalytic sequences
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
AU649074B2 (en) 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
CA2135646A1 (en) 1992-05-11 1993-11-25 Kenneth G. Draper Method and reagent for inhibiting viral replication
US6172208B1 (en) * 1992-07-06 2001-01-09 Genzyme Corporation Oligonucleotides modified with conjugate groups
JPH07509133A (ja) 1992-07-17 1995-10-12 リボザイム・ファーマシューティカルズ・インコーポレイテッド 動物疾患の処置のための方法および剤
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
US5591317A (en) 1994-02-16 1997-01-07 Pitts, Jr.; M. Michael Electrostatic device for water treatment
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
AU3889595A (en) 1994-10-05 1996-05-02 Amgen, Inc. Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein
US5783683A (en) 1995-01-10 1998-07-21 Genta Inc. Antisense oligonucleotides which reduce expression of the FGFRI gene
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
US5763263A (en) 1995-11-27 1998-06-09 Dehlinger; Peter J. Method and apparatus for producing position addressable combinatorial libraries
US5739119A (en) 1996-11-15 1998-04-14 Galli; Rachel L. Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA
CA2518475C (en) 2003-03-07 2014-12-23 Alnylam Pharmaceuticals, Inc. Irna agents comprising asymmetrical modifications
EP2669377A3 (en) 2003-04-17 2015-10-14 Alnylam Pharmaceuticals Inc. Modified iRNA agents
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
AU2006279906B2 (en) 2005-08-10 2012-05-10 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
EP2229186A2 (en) 2007-12-04 2010-09-22 Alnylam Pharmaceuticals Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
RU2582235C2 (ru) 2010-12-29 2016-04-20 Ф.Хоффманн-Ля Рош Аг Низкомолекулярные конъюгаты для внутриклеточной доставки нуклеиновых кислот
WO2012178173A1 (en) * 2011-06-24 2012-12-27 Centrose, Llc Extracellular targeted drug conjugates
US8577565B2 (en) 2011-12-16 2013-11-05 GM Global Technology Operations LLC Limiting branch pressure to a solenoid valve in a fluid circuit
EP4253395A3 (en) * 2012-08-06 2023-11-29 Alnylam Pharmaceuticals, Inc. Processes for the preparation of carbohydrate conjugated rna agents
WO2015006740A2 (en) * 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
EP3865576A1 (en) * 2014-12-15 2021-08-18 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
TWI912633B (zh) 2016-06-06 2026-01-21 美商愛羅海德製藥公司 5'-環-磷酸修飾之核苷酸
MX2019002075A (es) 2016-08-23 2019-07-01 Dicerna Pharmaceuticals Inc Composiciones que comprenden oligonucleotidos modificados reversiblemente, y sus usos.
WO2018045317A1 (en) 2016-09-02 2018-03-08 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
JP2021521140A (ja) * 2018-04-12 2021-08-26 ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. オリゴヌクレオチド組成物及びその使用方法

Also Published As

Publication number Publication date
US20260021188A1 (en) 2026-01-22
WO2023014938A1 (en) 2023-02-09
JP2024529067A (ja) 2024-08-01
EP4380946A1 (en) 2024-06-12
JP2026095525A (ja) 2026-06-11
JP7842850B2 (ja) 2026-04-08
AU2022324471A1 (en) 2024-02-15
US12465647B2 (en) 2025-11-11
US20230138928A1 (en) 2023-05-04
IL310547A (en) 2024-03-01
KR20240070519A (ko) 2024-05-21
CA3227661A1 (en) 2023-02-09
CN117980315A (zh) 2024-05-03

Similar Documents

Publication Publication Date Title
MA35109B1 (fr) Derives de nucleoside 2-substitues et procedes d'utilisation de ceux-ci pour le traitement de maladies virales
ZA202403388B (en) Small molecules for treatement of cancer
MX2007000028A (es) Derivados de nicotinamida y su uso como agentes terapeuticos.
BRPI0416212A (pt) derivado de piridazinona, composição farmacêutica, uso do derivado, método para o tratamento de um sujeito afligido com uma condição ou doença susceptìvel de melhora pela inibição de fosfodiesterase 4 e produto de combinação
PT1404347E (pt) Nucleosidos 4'-substituidos para o tratamento de doencas mediadas pelo virus da hepatite c
MEP25908A (bs) Derivati pirazolo-hinazolina, postupci za njihovo dobijanje i njihova upotreba kao inhibitor kinaze
ZA202408030B (en) Tricyclic heterocyclic derivatives, compositions and uses thereof
AU2003301020A8 (en) Isoquinolinone derivatives and their use as therapeutic agents
MY141939A (en) Pyridazine derivatives and their use as therapeutic agents
BRPI0407976A (pt) tetrahidroisoquinolinas substituìdas em 2,5 e 2,6 para uso como moduladores de 5-ht6
NO20060974L (no) Pyridazinderivater og deres anvendelse som terapeutiske midler
NZ580226A (en) Dimer compounds as inhibitors of iap
UY30438A1 (es) Derivados n-sustituidos de 5-halo-4-[2-alquil-1-sustituidos-1h-imidazol-5-il]pirimidin-2-amina, composiciones-procesos para su preparacion y usos
MA34604B1 (fr) Derives de la pyrazine en tant que bloqueurs de l'enac
ES2191754T3 (es) Fenil-dihidrobenzofuranos.
MA31142B1 (fr) Derives heteroaryles de pyrrolidinyl et piperdinyl cetones.
NO20080697L (no) Modifiserte farmasoytiske doseringsformer for frigivelse av syklooksygenase enzyminhibitor
ZA202200660B (en) Process for the preparation of ridinilazole and crystalline forms thereof
DK0828727T3 (da) Cyclohexyldihydrobenzofuraner
BR0103210A (pt) Derivados de azabicicloalcano e suas utilizações terapêuticas
BR0213037A (pt) Derivados de pirimidina
ATE366107T1 (de) Verwendung von sulfodehydroabietinsäure zur behandlung entzündlicher darmerkrankungen
ATE344249T1 (de) Pyrazinone, diese verbindungen enthaltende zusammenstellungen
UA81640C2 (ru) Противомикобактериальные соединения, способ их получения, фармацевтическая композиция на их основе
MX2025013094A (es) Conjugados basados en lipidos para la administracion sistemica en el sistema nervioso central, el sistema nervioso periferico y ocular