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
Links
- 238000000034 method Methods 0.000 title abstract 2
- 230000004568 DNA-binding Effects 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 102000004169 proteins and genes Human genes 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 title 1
- 101000768460 Homo sapiens Protein unc-13 homolog A Proteins 0.000 abstract 5
- 102100027901 Protein unc-13 homolog A Human genes 0.000 abstract 5
- 102100040347 TAR DNA-binding protein 43 Human genes 0.000 abstract 4
- 101710150875 TAR DNA-binding protein 43 Proteins 0.000 abstract 4
- 108020000948 Antisense Oligonucleotides Proteins 0.000 abstract 1
- 239000000074 antisense oligonucleotide Substances 0.000 abstract 1
- 238000012230 antisense oligonucleotides Methods 0.000 abstract 1
- 239000003112 inhibitor Substances 0.000 abstract 1
- 230000035772 mutation Effects 0.000 abstract 1
Classifications
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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)
- Peptides Or Proteins (AREA)
- Enzymes And Modification Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
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 (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)
| 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)
| 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 |
-
2022
- 2022-04-05 AU AU2022255175A patent/AU2022255175A1/en active Pending
- 2022-04-05 US US18/285,802 patent/US20250011773A1/en active Pending
- 2022-04-05 EP EP22718532.9A patent/EP4320236A1/en active Pending
- 2022-04-05 WO PCT/US2022/023559 patent/WO2022216759A1/en not_active Ceased
- 2022-04-05 MX MX2023011794A patent/MX2023011794A/es unknown
- 2022-04-05 IL IL307305A patent/IL307305A/en unknown
- 2022-04-05 CA CA3213590A patent/CA3213590A1/en active Pending
- 2022-04-05 KR KR1020237038205A patent/KR20240004467A/ko active Pending
- 2022-04-05 JP JP2023561290A patent/JP2024513237A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| IL307305A (en) | 2023-11-01 |
| US20250011773A1 (en) | 2025-01-09 |
| CA3213590A1 (en) | 2022-10-13 |
| EP4320236A1 (en) | 2024-02-14 |
| JP2024513237A (ja) | 2024-03-22 |
| KR20240004467A (ko) | 2024-01-11 |
| WO2022216759A1 (en) | 2022-10-13 |
| AU2022255175A1 (en) | 2023-11-23 |
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