AU2022402929A1 - Splice switcher antisense oligonucleotides with modified backbone chemistries - Google Patents

Splice switcher antisense oligonucleotides with modified backbone chemistries Download PDF

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Publication number
AU2022402929A1
AU2022402929A1 AU2022402929A AU2022402929A AU2022402929A1 AU 2022402929 A1 AU2022402929 A1 AU 2022402929A1 AU 2022402929 A AU2022402929 A AU 2022402929A AU 2022402929 A AU2022402929 A AU 2022402929A AU 2022402929 A1 AU2022402929 A1 AU 2022402929A1
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oligonucleotide
seq
linkage
nos
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AU2022402929A
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Duncan Brown
Daniel Elbaum
Sandra HINCKLEY
Marisa Elizabeth KAMELGARN
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Quralis Corp
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Quralis Corp
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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
    • A—HUMAN NECESSITIES
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    • 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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    • A—HUMAN NECESSITIES
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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2310/30—Chemical structure
    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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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
    • C12N2310/35—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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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
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AU2022402929A 2021-12-03 2022-12-02 Splice switcher antisense oligonucleotides with modified backbone chemistries Pending AU2022402929A1 (en)

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 (1)

Publication Number Publication Date
AU2022402929A1 true AU2022402929A1 (en) 2024-06-13

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AU2022402929A Pending AU2022402929A1 (en) 2021-12-03 2022-12-02 Splice switcher antisense oligonucleotides with modified backbone chemistries

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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

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EP4441226A2 (en) 2024-10-09
IL313141A (en) 2024-07-01
WO2023102242A2 (en) 2023-06-08
JP2024545052A (ja) 2024-12-05
KR20240129626A9 (ko) 2025-12-10
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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