ECSP23096498A - Restos de administración terapéutica novedosos y usos de estos - Google Patents

Restos de administración terapéutica novedosos y usos de estos

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Publication number
ECSP23096498A
ECSP23096498A ECSENADI202396498A ECDI202396498A ECSP23096498A EC SP23096498 A ECSP23096498 A EC SP23096498A EC SENADI202396498 A ECSENADI202396498 A EC SENADI202396498A EC DI202396498 A ECDI202396498 A EC DI202396498A EC SP23096498 A ECSP23096498 A EC SP23096498A
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EC
Ecuador
Prior art keywords
therapeutic administration
novel therapeutic
administration remains
remains
novel
Prior art date
Application number
ECSENADI202396498A
Other languages
English (en)
Inventor
Takako Wilson
Gregory Lawrence Lackner
Patrick Joseph Antonellis
Original Assignee
Lilly Co Eli
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 Lilly Co Eli filed Critical Lilly Co Eli
Publication of ECSP23096498A publication Critical patent/ECSP23096498A/es

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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 against enzymes
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
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    • C12N2310/3521Methyl
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
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    • C12N2330/00Production
    • C12N2330/30Production chemically synthesised

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente invención se relaciona con compuestos novedosos que comprenden restos de administración novedosos para la administración de oligonucleótidos, que son útiles en el tratamiento de enfermedades, especialmente enfermedades del hígado.
ECSENADI202396498A 2021-06-24 2023-12-22 Restos de administración terapéutica novedosos y usos de estos ECSP23096498A (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202163214555P 2021-06-24 2021-06-24

Publications (1)

Publication Number Publication Date
ECSP23096498A true ECSP23096498A (es) 2024-01-31

Family

ID=82595183

Family Applications (1)

Application Number Title Priority Date Filing Date
ECSENADI202396498A ECSP23096498A (es) 2021-06-24 2023-12-22 Restos de administración terapéutica novedosos y usos de estos

Country Status (17)

Country Link
US (1) US20240327839A1 (es)
EP (1) EP4359529A1 (es)
JP (2) JP2024522852A (es)
KR (1) KR20240023631A (es)
CN (1) CN117858948A (es)
AU (1) AU2022299173A1 (es)
CA (1) CA3224134A1 (es)
CL (1) CL2023003883A1 (es)
CO (1) CO2023018231A2 (es)
CR (1) CR20240003A (es)
DO (1) DOP2023000285A (es)
EC (1) ECSP23096498A (es)
IL (1) IL309503A (es)
JO (1) JOP20230326A1 (es)
MX (1) MX2024000208A (es)
PE (1) PE20250120A1 (es)
WO (1) WO2022271806A1 (es)

Families Citing this family (24)

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Publication number Priority date Publication date Assignee Title
GB2613225B (en) 2020-03-04 2024-01-03 Verve Therapeutics Inc Compositions and methods for targeted RNA delivery
WO2023015223A2 (en) * 2021-08-03 2023-02-09 Verve Therapeutics, Inc. Compositions and methods for targeted rna delivery
EP4479535A1 (en) 2022-02-14 2024-12-25 ProQR Therapeutics II B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
PE20242222A1 (es) 2022-03-14 2024-11-19 Lilly Co Eli Agentes de interferencia de arn de sarm1
WO2024110565A1 (en) 2022-11-24 2024-05-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis
WO2024175550A1 (en) 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
EP4688143A1 (en) 2023-03-27 2026-02-11 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of liver disease
WO2025049634A1 (en) * 2023-08-29 2025-03-06 Ganna Bio, Inc. Glycan conjugate compositions and methods
WO2025072748A2 (en) 2023-09-29 2025-04-03 Insitro, Inc. Compositions and methods for treating nonalcoholic fatty liver disease
CN121941769A (zh) 2023-10-02 2026-04-28 伊莱利利公司 Ppp1r15b rna干扰剂
WO2025104239A1 (en) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
WO2025155911A1 (en) 2024-01-18 2025-07-24 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hurler syndrome
WO2025188589A1 (en) 2024-03-04 2025-09-12 Eli Lilly And Company NOVEL NUCLEOTIDES AND OLIGONUCLEOTIDES AND RNAi AGENTS COMPRISING THE SAME
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
WO2025226980A2 (en) 2024-04-25 2025-10-30 Insitro, Inc. Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease
WO2025264948A2 (en) 2024-06-21 2025-12-26 Eli Lilly And Company Inhbe rna interference agents
WO2025264952A2 (en) 2024-06-21 2025-12-26 Eli Lilly And Company Novel rna therapeutics and uses thereof
WO2026006109A2 (en) 2024-06-24 2026-01-02 Eli Lilly And Company Novel rna therapeutics and uses thereof
WO2026006107A1 (en) 2024-06-24 2026-01-02 Eli Lilly And Company Novel oligonucleotides bearing a "2-propyl" internucleoside linkage
GB202410081D0 (en) 2024-07-11 2024-08-28 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of cardiovascular disease
WO2026044017A2 (en) 2024-08-21 2026-02-26 Insitro, Inc. Compositions and methods for treating nonalcoholic fatty liver disease
WO2026068781A1 (en) 2024-09-30 2026-04-02 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease

Family Cites Families (8)

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Publication number Priority date Publication date Assignee Title
DK2992098T3 (da) * 2013-05-01 2019-06-17 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af hbv- og ttr-ekspression
WO2019127004A1 (zh) * 2017-12-26 2019-07-04 广州市锐博生物科技有限公司 修饰的寡核苷酸和可用于合成修饰的寡核苷酸的化合物
KR102398295B1 (ko) * 2018-03-09 2022-05-17 다이이찌 산쿄 가부시키가이샤 당원병 Ia형 치료약
CN120247995A (zh) * 2018-05-30 2025-07-04 诺华股份有限公司 脂质修饰的核酸化合物和方法
CR20210395A (es) * 2018-12-21 2021-11-05 Ionis Pharmaceuticals Inc Moduladores de la expresión de hsd17b13
EP3923989A1 (en) * 2019-03-21 2021-12-22 Mitotherapeutix LLC Multivalent ligand clusters for targeted delivery of therapeutic agents
KR20220062517A (ko) * 2019-08-15 2022-05-17 아이오니스 파마수티컬즈, 인코포레이티드 결합 변형된 올리고머 화합물 및 이의 용도
US20220281911A1 (en) * 2019-08-19 2022-09-08 Mina Therapeutics Limited Oligonucleotide conjugate compositions and methods of use

Also Published As

Publication number Publication date
IL309503A (en) 2024-02-01
US20240327839A1 (en) 2024-10-03
CA3224134A1 (en) 2022-12-29
CR20240003A (es) 2024-01-25
CN117858948A (zh) 2024-04-09
MX2024000208A (es) 2024-01-29
JP2025176122A (ja) 2025-12-03
JP2024522852A (ja) 2024-06-21
EP4359529A1 (en) 2024-05-01
KR20240023631A (ko) 2024-02-22
WO2022271806A1 (en) 2022-12-29
JOP20230326A1 (ar) 2023-12-21
PE20250120A1 (es) 2025-01-16
CO2023018231A2 (es) 2024-01-25
CL2023003883A1 (es) 2024-11-15
DOP2023000285A (es) 2024-02-15
AU2022299173A1 (en) 2024-01-18

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