CA3239482A1 - Treatment of neurological diseases using modulators of unc13a gene transcripts - Google Patents

Treatment of neurological diseases using modulators of unc13a gene transcripts Download PDF

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Publication number
CA3239482A1
CA3239482A1 CA3239482A CA3239482A CA3239482A1 CA 3239482 A1 CA3239482 A1 CA 3239482A1 CA 3239482 A CA3239482 A CA 3239482A CA 3239482 A CA3239482 A CA 3239482A CA 3239482 A1 CA3239482 A1 CA 3239482A1
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oligonucleotide
linkage
seq
spacer
nos
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Pending
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CA3239482A
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English (en)
French (fr)
Inventor
Sandra HINCKLEY
Duncan Brown
Daniel Elbaum
Marisa Elizabeth KAMELGARN
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Quralis Corp
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Quralis Corp
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Publication of CA3239482A1 publication Critical patent/CA3239482A1/en
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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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • C12N2320/33—Alteration of splicing

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  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3239482A 2021-12-03 2022-12-02 Treatment of neurological diseases using modulators of unc13a gene transcripts Pending CA3239482A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163285786P 2021-12-03 2021-12-03
US63/285,786 2021-12-03
US202263350206P 2022-06-08 2022-06-08
US63/350,206 2022-06-08
US202263398987P 2022-08-18 2022-08-18
US63/398,987 2022-08-18
PCT/US2022/051713 WO2023102225A2 (en) 2021-12-03 2022-12-02 Treatment of neurological diseases using modulators of unc13a gene transcripts

Publications (1)

Publication Number Publication Date
CA3239482A1 true CA3239482A1 (en) 2023-06-08

Family

ID=86613080

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3239482A Pending CA3239482A1 (en) 2021-12-03 2022-12-02 Treatment of neurological diseases using modulators of unc13a gene transcripts

Country Status (16)

Country Link
US (1) US20250051766A1 (https=)
EP (1) EP4441225A4 (https=)
JP (1) JP2024543214A (https=)
KR (1) KR20240129627A9 (https=)
CN (1) CN119790152A (https=)
AU (1) AU2022400851A1 (https=)
CA (1) CA3239482A1 (https=)
CL (1) CL2024001657A1 (https=)
CO (1) CO2024008076A2 (https=)
CR (1) CR20240269A (https=)
DO (1) DOP2024000107A (https=)
IL (1) IL313203A (https=)
JO (1) JOP20240125A1 (https=)
MX (1) MX2024006763A (https=)
PE (1) PE20250677A1 (https=)
WO (1) WO2023102225A2 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
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
WO2025186619A1 (en) * 2024-03-05 2025-09-12 Takeda Pharmaceutical Company Limited Compositions and methods for splicing modulation of unc13a
WO2025255388A1 (en) * 2024-06-05 2025-12-11 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas
WO2026080323A1 (en) 2024-10-09 2026-04-16 Quralis Corporation Treatment of neurological diseases using modulators of unc13a gene transcripts

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060008800A1 (en) * 2001-11-05 2006-01-12 Christian Rosenmund Unc-13 in the modulation of neurotransmission and secretion events
AU2013203395A1 (en) * 2012-03-30 2013-10-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome
DK3155101T3 (da) * 2014-06-16 2020-05-04 Univ Johns Hopkins Sammensætninger og fremgangsmåder til ekspression af CRISPR-leder-RNA´er under anvendelse af H1-promotoren
WO2020102472A1 (en) * 2018-11-15 2020-05-22 President And Fellows Of Harvard College Methods and compositions related to targeting ffar2 and ilc3 populations for the treatment of a gastrointestinal disease
AU2022255175A1 (en) * 2021-04-06 2023-11-23 Trace Neuroscience, Inc. Compositions and methods for treating tdp-43 proteinopathy

Also Published As

Publication number Publication date
EP4441225A2 (en) 2024-10-09
AU2022400851A1 (en) 2024-06-20
IL313203A (en) 2024-07-01
EP4441225A4 (en) 2026-03-11
WO2023102225A2 (en) 2023-06-08
JOP20240125A1 (ar) 2024-06-02
CR20240269A (es) 2024-09-13
CN119790152A (zh) 2025-04-08
CO2024008076A2 (es) 2024-11-18
WO2023102225A3 (en) 2024-03-28
US20250051766A1 (en) 2025-02-13
KR20240129627A (ko) 2024-08-27
MX2024006763A (es) 2024-08-15
CL2024001657A1 (es) 2024-12-20
PE20250677A1 (es) 2025-03-04
KR20240129627A9 (ko) 2025-12-10
JP2024543214A (ja) 2024-11-19
DOP2024000107A (es) 2024-09-30

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