MX2024003612A - Composiciones de oligonucleotidos y metodos de estas. - Google Patents

Composiciones de oligonucleotidos y metodos de estas.

Info

Publication number
MX2024003612A
MX2024003612A MX2024003612A MX2024003612A MX2024003612A MX 2024003612 A MX2024003612 A MX 2024003612A MX 2024003612 A MX2024003612 A MX 2024003612A MX 2024003612 A MX2024003612 A MX 2024003612A MX 2024003612 A MX2024003612 A MX 2024003612A
Authority
MX
Mexico
Prior art keywords
present disclosure
compositions
oligonucleotides
methods
oligonucleotide compositions
Prior art date
Application number
MX2024003612A
Other languages
English (en)
Inventor
Chandra Vargeese
Naoki Iwamoto
Hui Yu
Genliang Lu
Hailin Yang
Chikdu Shakti Shivalila
Mamoru Shimizu
Prashant Monian
Pachamuthu Kandasamy
Paloma Hoban Giangrande
Sarah Diane Lamore
Fengjiao Zhang
Padmakumar Narayanan
Original Assignee
Wave Life Sciences Ltd
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
Priority claimed from PCT/US2021/058495 external-priority patent/WO2022099159A1/en
Application filed by Wave Life Sciences Ltd filed Critical Wave Life Sciences Ltd
Publication of MX2024003612A publication Critical patent/MX2024003612A/es

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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

Abstract

Entre otras cosas, la presente divulgación proporciona oligonucleótidos diseñados y composiciones de estos. En algunas realizaciones, los oligonucleótidos y composiciones de la presente divulgación pueden proporcionar niveles elevados de edición de adenosina. En algunas realizaciones, los oligonucleótidos y composiciones de la presente divulgación son útiles para tratar diversas afecciones, trastornos o enfermedades, p. ej., la deficiencia de alfa-1 antitripsina. En algunas realizaciones, la presente divulgación proporciona métodos para tratar diversas afecciones, trastornos o enfermedades que pueden beneficiarse de la edición de adenosina.
MX2024003612A 2021-09-26 2022-09-26 Composiciones de oligonucleotidos y metodos de estas. MX2024003612A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163248520P 2021-09-26 2021-09-26
PCT/US2021/058495 WO2022099159A1 (en) 2020-11-08 2021-11-08 Oligonucleotide compositions and methods thereof
US202263331756P 2022-04-15 2022-04-15
US202263397320P 2022-08-11 2022-08-11
PCT/US2022/044765 WO2023049475A1 (en) 2021-09-26 2022-09-26 Oligonucleotide compositions and methods thereof

Publications (1)

Publication Number Publication Date
MX2024003612A true MX2024003612A (es) 2024-05-16

Family

ID=85721200

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024003612A MX2024003612A (es) 2021-09-26 2022-09-26 Composiciones de oligonucleotidos y metodos de estas.

Country Status (9)

Country Link
EP (1) EP4396354A1 (es)
JP (1) JP2024535388A (es)
KR (1) KR20240063964A (es)
AU (1) AU2022352779A1 (es)
CA (1) CA3232070A1 (es)
CO (1) CO2024005352A2 (es)
IL (1) IL311625A (es)
MX (1) MX2024003612A (es)
WO (1) WO2023049475A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11873316B2 (en) 2016-11-23 2024-01-16 Wave Life Sciences Ltd. Compositions and methods for phosphoramidite and oligonucleotide synthesis

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100048680A1 (en) * 2007-01-11 2010-02-25 University Of Florida Research Foundation Inc. Alpha-1-Antitrypsin Variants and Uses Thereof
MA45270A (fr) * 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
KR20210126680A (ko) * 2019-02-13 2021-10-20 빔 테라퓨틱스, 인크. 알파-1 항트립신 결핍증을 치료하기 위한 조성물 및 방법

Also Published As

Publication number Publication date
IL311625A (en) 2024-05-01
CA3232070A1 (en) 2023-03-30
AU2022352779A1 (en) 2024-03-28
JP2024535388A (ja) 2024-09-30
WO2023049475A1 (en) 2023-03-30
KR20240063964A (ko) 2024-05-10
EP4396354A1 (en) 2024-07-10
CO2024005352A2 (es) 2024-05-30

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