MX2024006612A - Oligonucleotidos antisentido de cambio de empalme con estructuras quimicas modificadas. - Google Patents

Oligonucleotidos antisentido de cambio de empalme con estructuras quimicas modificadas.

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Publication number
MX2024006612A
MX2024006612A MX2024006612A MX2024006612A MX2024006612A MX 2024006612 A MX2024006612 A MX 2024006612A MX 2024006612 A MX2024006612 A MX 2024006612A MX 2024006612 A MX2024006612 A MX 2024006612A MX 2024006612 A MX2024006612 A MX 2024006612A
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MX
Mexico
Prior art keywords
unc13a
stmn2
kcnq2
transcripts
switcher
Prior art date
Application number
MX2024006612A
Other languages
English (en)
Spanish (es)
Inventor
Daniel Elbaum
Duncan Brown
Sandra Hinckley
Marisa Elizabeth Kamelgarn
Original Assignee
Quralis Corp
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
Application filed by Quralis Corp filed Critical Quralis Corp
Publication of MX2024006612A publication Critical patent/MX2024006612A/es

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07H1/02Phosphorylation
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    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides 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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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/33Chemical structure of the base
    • C12N2310/332Abasic residue
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
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    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

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  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Engineering & Computer Science (AREA)
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  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • Veterinary Medicine (AREA)
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  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
MX2024006612A 2021-12-03 2022-12-02 Oligonucleotidos antisentido de cambio de empalme con estructuras quimicas modificadas. MX2024006612A (es)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US202163285628P 2021-12-03 2021-12-03
US202163285786P 2021-12-03 2021-12-03
US202163285631P 2021-12-03 2021-12-03
US202163285933P 2021-12-03 2021-12-03
US202263350206P 2022-06-08 2022-06-08
US202263398992P 2022-08-18 2022-08-18
US202263398987P 2022-08-18 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
MX2024006612A true MX2024006612A (es) 2024-06-12

Family

ID=86613046

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024006612A MX2024006612A (es) 2021-12-03 2022-12-02 Oligonucleotidos antisentido de cambio de empalme con estructuras quimicas modificadas.

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

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Publication number Publication date
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
AU2022402929A1 (en) 2024-06-13
CA3241870A1 (en) 2023-06-08
WO2023102242A3 (en) 2023-10-12
WO2023102242A9 (en) 2024-10-31
KR20240129626A (ko) 2024-08-27
CN119522282A (zh) 2025-02-25

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