AU2022255175A1 - Compositions and methods for treating tdp-43 proteinopathy - Google Patents

Compositions and methods for treating tdp-43 proteinopathy Download PDF

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Publication number
AU2022255175A1
AU2022255175A1 AU2022255175A AU2022255175A AU2022255175A1 AU 2022255175 A1 AU2022255175 A1 AU 2022255175A1 AU 2022255175 A AU2022255175 A AU 2022255175A AU 2022255175 A AU2022255175 A AU 2022255175A AU 2022255175 A1 AU2022255175 A1 AU 2022255175A1
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AU
Australia
Prior art keywords
seq
antisense oligonucleotide
bases
unc13a
exon
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Pending
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AU2022255175A
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English (en)
Inventor
Eric Green
Shila MEKHOUBAD
Georgiana MILLER
Nathan SALLEE
David Wyatt
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Trace Neuroscience Inc
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Trace Neuroscience Inc
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Publication of AU2022255175A1 publication Critical patent/AU2022255175A1/en
Assigned to Trace NewCo, Inc. reassignment Trace NewCo, Inc. Request for Assignment Assignors: MAZE THERAPEUTICS, INC.
Assigned to TRACE NEUROSCIENCE, INC. reassignment TRACE NEUROSCIENCE, INC. Amend patent request/document other than specification (104) Assignors: Trace NewCo, Inc.
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2022255175A 2021-04-06 2022-04-05 Compositions and methods for treating tdp-43 proteinopathy Pending AU2022255175A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163171522P 2021-04-06 2021-04-06
US63/171,522 2021-04-06
US202263312808P 2022-02-22 2022-02-22
US63/312,808 2022-02-22
PCT/US2022/023559 WO2022216759A1 (en) 2021-04-06 2022-04-05 Compositions and methods for treating tdp-43 proteinopathy

Publications (1)

Publication Number Publication Date
AU2022255175A1 true AU2022255175A1 (en) 2023-11-23

Family

ID=81384958

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2022255175A Pending AU2022255175A1 (en) 2021-04-06 2022-04-05 Compositions and methods for treating tdp-43 proteinopathy

Country Status (9)

Country Link
US (1) US20250011773A1 (https=)
EP (1) EP4320236A1 (https=)
JP (1) JP2024513237A (https=)
KR (1) KR20240004467A (https=)
AU (1) AU2022255175A1 (https=)
CA (1) CA3213590A1 (https=)
IL (1) IL307305A (https=)
MX (1) MX2023011794A (https=)
WO (1) WO2022216759A1 (https=)

Families Citing this family (13)

* 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
CA3227115A1 (en) * 2021-07-21 2023-01-26 Wen-Hsuan Chang Unc13a antisense oligonucleotides
CN119790152A (zh) * 2021-12-03 2025-04-08 奎里斯公司 使用unc13a基因转录本的调节剂治疗神经系统疾病
GB202117758D0 (en) * 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
CN118974254A (zh) * 2021-12-21 2024-11-15 豪夫迈·罗氏有限公司 靶向unc13a的反义寡核苷酸
AU2023356317A1 (en) * 2022-10-05 2025-05-15 Trace Neuroscience, Inc. Unc13a antisense oligonucleotides and uses thereof
WO2024155986A2 (en) * 2023-01-20 2024-07-25 AcuraStem Incorporated Unc13a antisense oligonucleotides
WO2024178223A1 (en) * 2023-02-24 2024-08-29 Northwestern University Antisense oligonucleotides for preventing unc13a misplicing
AU2024283557A1 (en) * 2023-06-02 2026-01-08 Quralis Corporation Modified unc13a oligonucleotides
GB202309922D0 (en) * 2023-06-29 2023-08-16 Univ Edinburgh Antisense treatment
WO2025007194A1 (en) * 2023-07-05 2025-01-09 Macquarie University Modulation of gene expression
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 (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016528873A (ja) * 2012-05-16 2016-09-23 ラナ セラピューティクス インコーポレイテッド 遺伝子発現を調節するための組成物及び方法
ES2762326T5 (es) 2012-10-15 2023-04-27 Ionis Pharmaceuticals Inc Métodos para modular la expresión de C9ORF72
EP4166667A3 (en) 2013-10-11 2023-08-02 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
EP4410805A3 (en) 2014-03-18 2024-11-27 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
BR112018012894A2 (en) 2015-12-23 2018-12-04 Crispr Therapeutics Ag Materials and Methods for Treatment of Amyotrophic Lateral Sclerosis and / or Frontotemporal Lobular Degeneration
TW201920672A (zh) 2017-08-08 2019-06-01 新加坡商波濤生命科學有限公司 寡核苷酸組合物及其使用方法
CA3079727A1 (en) 2017-10-24 2019-05-02 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of rare diseases
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
WO2022018187A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Oligonucleotides targeting rna binding protein sites
GB2603454A (en) * 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders

Also Published As

Publication number Publication date
IL307305A (en) 2023-11-01
US20250011773A1 (en) 2025-01-09
CA3213590A1 (en) 2022-10-13
MX2023011794A (es) 2024-01-08
EP4320236A1 (en) 2024-02-14
JP2024513237A (ja) 2024-03-22
KR20240004467A (ko) 2024-01-11
WO2022216759A1 (en) 2022-10-13

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Owner name: TRACE NEWCO, INC.

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