CO2024005352A2 - Composiciones de oligonucleótidos y métodos de estas - Google Patents

Composiciones de oligonucleótidos y métodos de estas

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Publication number
CO2024005352A2
CO2024005352A2 CONC2024/0005352A CO2024005352A CO2024005352A2 CO 2024005352 A2 CO2024005352 A2 CO 2024005352A2 CO 2024005352 A CO2024005352 A CO 2024005352A CO 2024005352 A2 CO2024005352 A2 CO 2024005352A2
Authority
CO
Colombia
Prior art keywords
present disclosure
compositions
oligonucleotides
methods
oligonucleotide compositions
Prior art date
Application number
CONC2024/0005352A
Other languages
English (en)
Inventor
Chandra Vargeese
Naoki Iwamoto
Genliang Lu
Hailin Yang
Prashant Monian
Chikdu Shakti Shivalila
Paloma Hoban Giangrande
Pachamuthu Kandasamy
Mamoru Shimizu
Hui Yu
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 CO2024005352A2 publication Critical patent/CO2024005352A2/es

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    • 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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/35—Nature of the modification
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    • 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
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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)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (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.
CONC2024/0005352A 2021-09-26 2024-05-07 Composiciones de oligonucleótidos y métodos de estas CO2024005352A2 (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
CO2024005352A2 true CO2024005352A2 (es) 2024-05-30

Family

ID=85721200

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2024/0005352A CO2024005352A2 (es) 2021-09-26 2024-05-07 Composiciones de oligonucleótidos y métodos de estas

Country Status (7)

Country Link
EP (1) EP4396354A1 (es)
KR (1) KR20240063964A (es)
AU (1) AU2022352779A1 (es)
CA (1) CA3232070A1 (es)
CO (1) CO2024005352A2 (es)
IL (1) IL311625A (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
MA46905A (fr) 2016-11-23 2019-10-02 Wave Life Sciences Ltd Compositions et procédés de synthèse de phosphoramidites et d'oligonucléotides

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
CN114072180A (zh) * 2019-02-13 2022-02-18 比姆医疗股份有限公司 用于治疗α1-抗胰蛋白酶缺乏症的组合物和方法

Also Published As

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

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