KR20240129626A9 - 변형된 백본 화학을 갖는 스플라이스 스위처 안티센스 올리고뉴클레오티드 - Google Patents

변형된 백본 화학을 갖는 스플라이스 스위처 안티센스 올리고뉴클레오티드

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Publication number
KR20240129626A9
KR20240129626A9 KR1020247021691A KR20247021691A KR20240129626A9 KR 20240129626 A9 KR20240129626 A9 KR 20240129626A9 KR 1020247021691 A KR1020247021691 A KR 1020247021691A KR 20247021691 A KR20247021691 A KR 20247021691A KR 20240129626 A9 KR20240129626 A9 KR 20240129626A9
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Prior art keywords
switcher
splice
antisense oligonucleotides
modified backbone
backbone chemistry
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Pending
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KR1020247021691A
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English (en)
Korean (ko)
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KR20240129626A (ko
Inventor
산드라 힝클리
던컨 브라운
다니엘 엘바움
마리사 엘리자베스 카멜가른
Original Assignee
큐랄리스 코포레이션
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Publication of KR20240129626A publication Critical patent/KR20240129626A/ko
Publication of KR20240129626A9 publication Critical patent/KR20240129626A9/ko
Pending legal-status Critical Current

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
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    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/30—Chemical structure
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    • C12N2310/35—Nature of the modification
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
KR1020247021691A 2021-12-03 2022-12-02 변형된 백본 화학을 갖는 스플라이스 스위처 안티센스 올리고뉴클레오티드 Pending KR20240129626A9 (ko)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US202163285628P 2021-12-03 2021-12-03
US202163285933P 2021-12-03 2021-12-03
US202163285786P 2021-12-03 2021-12-03
US202163285631P 2021-12-03 2021-12-03
US63/285,631 2021-12-03
US63/285,933 2021-12-03
US63/285,786 2021-12-03
US63/285,628 2021-12-03
US202263350206P 2022-06-08 2022-06-08
US63/350,206 2022-06-08
US202263398987P 2022-08-18 2022-08-18
US202263398992P 2022-08-18 2022-08-18
US63/398,992 2022-08-18
US63/398,987 2022-08-18
PCT/US2022/051740 WO2023102242A2 (en) 2021-12-03 2022-12-02 Splice switcher antisense oligonucleotides with modified backbone chemistries

Publications (2)

Publication Number Publication Date
KR20240129626A KR20240129626A (ko) 2024-08-27
KR20240129626A9 true KR20240129626A9 (ko) 2025-12-10

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KR1020247021691A Pending KR20240129626A9 (ko) 2021-12-03 2022-12-02 변형된 백본 화학을 갖는 스플라이스 스위처 안티센스 올리고뉴클레오티드

Country Status (9)

Country Link
EP (1) EP4441226A2 (https=)
JP (1) JP2024545052A (https=)
KR (1) KR20240129626A9 (https=)
CN (1) CN119522282A (https=)
AU (1) AU2022402929A1 (https=)
CA (1) CA3241870A1 (https=)
IL (1) IL313141A (https=)
MX (1) MX2024006612A (https=)
WO (1) WO2023102242A2 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023356317A1 (en) * 2022-10-05 2025-05-15 Trace Neuroscience, Inc. Unc13a antisense oligonucleotides and uses thereof
WO2024178223A1 (en) * 2023-02-24 2024-08-29 Northwestern University Antisense oligonucleotides for preventing unc13a misplicing
GB202315883D0 (en) * 2023-10-17 2023-11-29 Univ Oxford Innovation Ltd Method
WO2025186619A1 (en) * 2024-03-05 2025-09-12 Takeda Pharmaceutical Company Limited Compositions and methods for splicing modulation of unc13a

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003232071A1 (en) * 2002-05-06 2003-11-17 Genelabs Technologies, Inc. Nucleoside derivatives for treating hepatitis c virus infection
EP3260540A1 (en) * 2010-11-12 2017-12-27 The General Hospital Corporation Polycomb-associated non-coding rnas
WO2020150290A2 (en) * 2019-01-14 2020-07-23 President And Fellows Of Harvard College Methods and compositions for restoring stmn2 levels
JP7795914B2 (ja) * 2019-06-03 2026-01-08 クラリス コーポレーション 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法

Also Published As

Publication number Publication date
EP4441226A2 (en) 2024-10-09
IL313141A (en) 2024-07-01
WO2023102242A2 (en) 2023-06-08
JP2024545052A (ja) 2024-12-05
AU2022402929A1 (en) 2024-06-13
CA3241870A1 (en) 2023-06-08
MX2024006612A (es) 2024-06-12
WO2023102242A3 (en) 2023-10-12
WO2023102242A9 (en) 2024-10-31
KR20240129626A (ko) 2024-08-27
CN119522282A (zh) 2025-02-25

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