CO2023018002A2 - Compuestos antisentido y métodos para dirigirse a repeticiones cug - Google Patents
Compuestos antisentido y métodos para dirigirse a repeticiones cugInfo
- Publication number
- CO2023018002A2 CO2023018002A2 CONC2023/0018002A CO2023018002A CO2023018002A2 CO 2023018002 A2 CO2023018002 A2 CO 2023018002A2 CO 2023018002 A CO2023018002 A CO 2023018002A CO 2023018002 A2 CO2023018002 A2 CO 2023018002A2
- Authority
- CO
- Colombia
- Prior art keywords
- methods
- antisense compounds
- cug repeats
- expanded
- compounds
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 5
- 230000000692 anti-sense effect Effects 0.000 title abstract 3
- 230000008685 targeting Effects 0.000 title 1
- 101000701517 Homo sapiens Putative protein ATXN8OS Proteins 0.000 abstract 2
- 102100030469 Putative protein ATXN8OS Human genes 0.000 abstract 2
- 201000010099 disease Diseases 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 201000009340 myotonic dystrophy type 1 Diseases 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 abstract 1
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 abstract 1
- 108010069514 Cyclic Peptides Proteins 0.000 abstract 1
- 102000001189 Cyclic Peptides Human genes 0.000 abstract 1
- 208000037140 Steinert myotonic dystrophy Diseases 0.000 abstract 1
- 125000004122 cyclic group Chemical group 0.000 abstract 1
Classifications
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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- 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
- A61K47/62—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 a protein, peptide or polyamino acid
- A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
- A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
- A61K47/6455—Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
- C07K7/64—Cyclic peptides containing only normal peptide links
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- 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
- C12N2310/11—Antisense
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- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2310/3513—Protein; Peptide
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Neurosurgery (AREA)
- Immunology (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Abstract
Se proporcionan compuestos que comprenden un péptido cíclico, tal como un péptido penetrante de células cíclico, y un compuesto antisentido. El compuesto antisentido se une a un gen que tiene una repetición CTG expandida o un transcrito génico que tiene una repetición CUG expandida. Los compuestos pueden suministrarse a los sujetos para tratar enfermedades asociadas con repeticiones CTG·CUG expandidas, tales como la distrofia miotónica tipo 1 (DM1), la ataxia espinocerebelosa-8 (SCA8) y la enfermedad similar a Huntington-2 (HDL2).
Applications Claiming Priority (16)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202163213900P | 2021-06-23 | 2021-06-23 | |
US202163239847P | 2021-09-01 | 2021-09-01 | |
US202163239671P | 2021-09-01 | 2021-09-01 | |
US202163290960P | 2021-12-17 | 2021-12-17 | |
US202163290892P | 2021-12-17 | 2021-12-17 | |
US202263298565P | 2022-01-11 | 2022-01-11 | |
US202263305071P | 2022-01-31 | 2022-01-31 | |
US202263268577P | 2022-02-25 | 2022-02-25 | |
US202263314369P | 2022-02-26 | 2022-02-26 | |
US202263316634P | 2022-03-04 | 2022-03-04 | |
US202263317856P | 2022-03-08 | 2022-03-08 | |
US202263362295P | 2022-03-31 | 2022-03-31 | |
US202263326201P | 2022-03-31 | 2022-03-31 | |
US202263327179P | 2022-04-04 | 2022-04-04 | |
US202263339250P | 2022-05-06 | 2022-05-06 | |
PCT/US2022/034517 WO2022271818A1 (en) | 2021-06-23 | 2022-06-22 | Antisense compounds and methods for targeting cug repeats |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2023018002A2 true CO2023018002A2 (es) | 2024-02-05 |
Family
ID=82608300
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2023/0018002A CO2023018002A2 (es) | 2021-06-23 | 2024-01-09 | Compuestos antisentido y métodos para dirigirse a repeticiones cug |
Country Status (7)
Country | Link |
---|---|
EP (1) | EP4359006A1 (es) |
KR (1) | KR20240038967A (es) |
AU (1) | AU2022298774A1 (es) |
CA (1) | CA3222824A1 (es) |
CO (1) | CO2023018002A2 (es) |
IL (1) | IL309001A (es) |
WO (1) | WO2022271818A1 (es) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2023219933A1 (en) * | 2022-05-09 | 2023-11-16 | Entrada Therapeutics, Inc. | Compositions and methods for delivery of nucleic acid therapeutics |
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CA2932472A1 (en) | 2013-12-12 | 2015-06-18 | Massachusetts Institute Of Technology | Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders |
EP3289076B1 (en) | 2015-05-01 | 2021-11-17 | Precision Biosciences, Inc. | Precise deletion of chromoscomal sequences in vivo |
AU2016335032B2 (en) | 2015-10-05 | 2022-04-14 | Proqr Therapeutics Ii B.V. | Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion |
JP6657520B2 (ja) | 2016-02-03 | 2020-03-04 | 株式会社神戸製鋼所 | タイヤ試験装置のタイヤ空気充填機構及びタイヤ空気充填方法 |
KR20170130253A (ko) | 2016-05-18 | 2017-11-28 | 에스케이텔레콤 주식회사 | 적응형 스트리밍 서비스 제공 방법 및 이를 위한 장치 |
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CN110139927A (zh) | 2016-11-16 | 2019-08-16 | 加利福尼亚大学董事会 | Crispr-cas9抑制剂 |
KR20180102871A (ko) | 2017-03-08 | 2018-09-18 | 엘지전자 주식회사 | 이동단말기 및 이동단말기의 차량 제어 방법 |
EP3592365A4 (en) | 2017-03-10 | 2021-01-13 | The Board Of Regents Of The University Of Texas System | TREATMENT OF ENDOTHELIAL CORNEAL DYSTROPHY OF FUCHS |
EP3755351A4 (en) * | 2018-02-22 | 2021-11-24 | Entrada Therapeutics, Inc. | COMPOSITIONS AND TREATMENT METHODS FOR MITOCHONDRIAL NEUROGASTROINTESTINAL ENCEPHALOPATHY |
GB201812972D0 (en) * | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
EP4058032A4 (en) * | 2019-12-19 | 2024-01-10 | Entrada Therapeutics Inc | COMPOSITIONS FOR ADMINISTRATION OF ANTISENSE COMPOUNDS |
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2022
- 2022-06-22 KR KR1020247002577A patent/KR20240038967A/ko unknown
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2024
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KR20240038967A (ko) | 2024-03-26 |
AU2022298774A1 (en) | 2023-12-14 |
CA3222824A1 (en) | 2022-12-29 |
IL309001A (en) | 2024-02-01 |
WO2022271818A1 (en) | 2022-12-29 |
EP4359006A1 (en) | 2024-05-01 |
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