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MX2023011794A - Composiciones y métodos para el tratamiento de la proteinopatía de proteina de union a acido desoxirribonucleico tar 43 (tdp-43). - Google Patents

Composiciones y métodos para el tratamiento de la proteinopatía de proteina de union a acido desoxirribonucleico tar 43 (tdp-43).

Info

Publication number
MX2023011794A
MX2023011794A MX2023011794A MX2023011794A MX2023011794A MX 2023011794 A MX2023011794 A MX 2023011794A MX 2023011794 A MX2023011794 A MX 2023011794A MX 2023011794 A MX2023011794 A MX 2023011794A MX 2023011794 A MX2023011794 A MX 2023011794A
Authority
MX
Mexico
Prior art keywords
tdp
tar
binding protein
unc13a
proteinopathy
Prior art date
Application number
MX2023011794A
Other languages
English (en)
Inventor
David Wyatt
Eric Green
Shila Mekhoubad
Georgiana Miller
Nathan Sallee
Original Assignee
Maze Therapeutics Inc
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 Maze Therapeutics Inc filed Critical Maze Therapeutics Inc
Publication of MX2023011794A publication Critical patent/MX2023011794A/es

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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

Landscapes

  • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La presente descripción se refiere al uso de inhibidores específicos de la variante de empalme de exón críptico de UNC13A en métodos para la reducción de la expresión de una variante de empalme de exón críptico de UNC13A en una célula, reducir la proteína 43 de unión a TAR ADN (TDP-43) fosforilada en una célula, tratar la proteinopatía por proteína 43 de unión a TAR ADN (TDP-43) en un sujeto o tratar un sujeto en el que se ha identificado una mutación de UNC13A en el intrón 20-21 de UNC13A. También se contemplan oligonucleótidos antisentido dirigidos a una variante de empalme críptica de UNC13A.
MX2023011794A 2021-04-06 2022-04-05 Composiciones y métodos para el tratamiento de la proteinopatía de proteina de union a acido desoxirribonucleico tar 43 (tdp-43). MX2023011794A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163171522P 2021-04-06 2021-04-06
US202263312808P 2022-02-22 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
MX2023011794A true MX2023011794A (es) 2024-01-08

Family

ID=81384958

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023011794A MX2023011794A (es) 2021-04-06 2022-04-05 Composiciones y métodos para el tratamiento de la proteinopatía de proteina de union a acido desoxirribonucleico tar 43 (tdp-43).

Country Status (9)

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

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2022316149A1 (en) 2021-07-21 2024-02-08 AcuraStem Incorporated Unc13a antisense oligonucleotides
WO2023102225A2 (en) * 2021-12-03 2023-06-08 Quralis Corporation Treatment of neurological diseases using modulators of unc13a gene transcripts
GB202117758D0 (en) * 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
WO2023118087A1 (en) * 2021-12-21 2023-06-29 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting unc13a
EP4599062A1 (en) * 2022-10-05 2025-08-13 Trace Neuroscience, Inc. Unc13a antisense oligonucleotides and uses thereof
EP4652276A2 (en) * 2023-01-20 2025-11-26 AcuraStem Incorporated Unc13a antisense oligonucleotides
WO2024178223A1 (en) * 2023-02-24 2024-08-29 Northwestern University Antisense oligonucleotides for preventing unc13a misplicing
WO2024249791A2 (en) * 2023-06-02 2024-12-05 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
WO2013173635A1 (en) * 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
DK2906696T4 (da) 2012-10-15 2023-02-27 Ionis Pharmaceuticals Inc Fremgangsmåder til modulering af c9orf72-ekspression
EP3055414A4 (en) 2013-10-11 2017-07-19 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
AU2015231294B2 (en) 2014-03-18 2020-10-29 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
CN109312339B (zh) 2015-12-23 2022-01-28 克里斯珀医疗股份公司 用于治疗肌萎缩侧索硬化症和/或额颞叶变性的材料和方法
WO2019032612A1 (en) 2017-08-08 2019-02-14 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED METHODS
US20190167815A1 (en) 2017-10-24 2019-06-06 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of rare diseases
US20220333105A1 (en) * 2019-06-03 2022-10-20 Quralis Corporation Oligonucleotides and methods of use for treating neurological diseases
EP4185696A1 (en) * 2020-07-23 2023-05-31 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
WO2022216759A1 (en) 2022-10-13
IL307305A (en) 2023-11-01
KR20240004467A (ko) 2024-01-11
EP4320236A1 (en) 2024-02-14
JP2024513237A (ja) 2024-03-22
CA3213590A1 (en) 2022-10-13
US20250011773A1 (en) 2025-01-09
AU2022255175A1 (en) 2023-11-23

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