MX2021014868A - Oligonucleotidos y metodos de uso para el tratamiento de enfermedades neurologicas. - Google Patents
Oligonucleotidos y metodos de uso para el tratamiento de enfermedades neurologicas.Info
- Publication number
- MX2021014868A MX2021014868A MX2021014868A MX2021014868A MX2021014868A MX 2021014868 A MX2021014868 A MX 2021014868A MX 2021014868 A MX2021014868 A MX 2021014868A MX 2021014868 A MX2021014868 A MX 2021014868A MX 2021014868 A MX2021014868 A MX 2021014868A
- Authority
- MX
- Mexico
- Prior art keywords
- methods
- oligonucleotides
- neurological diseases
- treatment
- diseases
- Prior art date
Links
Classifications
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- 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
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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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
-
- 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
-
- 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
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
-
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
- C12N2310/35—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
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
-
- 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
- C12N2320/31—Combination therapy
-
- 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
-
- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
- Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
- Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
- Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Nutrition Science (AREA)
- Physiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
- Steroid Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962856264P | 2019-06-03 | 2019-06-03 | |
| US201962914252P | 2019-10-11 | 2019-10-11 | |
| US201962949817P | 2019-12-18 | 2019-12-18 | |
| PCT/US2020/035811 WO2020247419A2 (en) | 2019-06-03 | 2020-06-03 | Oligonucleotides and methods of use for treating neurological diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021014868A true MX2021014868A (es) | 2022-03-25 |
Family
ID=73653367
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021014868A MX2021014868A (es) | 2019-06-03 | 2020-06-03 | Oligonucleotidos y metodos de uso para el tratamiento de enfermedades neurologicas. |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20220333105A1 (https=) |
| EP (1) | EP3976010A4 (https=) |
| JP (2) | JP7795914B2 (https=) |
| KR (1) | KR20220033472A (https=) |
| CN (1) | CN114555069A (https=) |
| AU (1) | AU2020288555A1 (https=) |
| BR (1) | BR112021024463A2 (https=) |
| CA (1) | CA3142526A1 (https=) |
| IL (1) | IL288574A (https=) |
| MX (1) | MX2021014868A (https=) |
| PH (1) | PH12021553024A1 (https=) |
| WO (1) | WO2020247419A2 (https=) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020150290A2 (en) | 2019-01-14 | 2020-07-23 | President And Fellows Of Harvard College | Methods and compositions for restoring stmn2 levels |
| EP4127172A4 (en) * | 2020-03-25 | 2025-06-04 | President and Fellows of Harvard College | Methods and compositions for restoring STMN2 levels |
| EP4162051A4 (en) * | 2020-06-03 | 2025-07-09 | Quralis Corp | TREATMENT OF NEUROLOGICAL DISEASES USING GENE TRANSCRIPT MODULATORS |
| WO2022018155A1 (en) * | 2020-07-23 | 2022-01-27 | F. Hoffmann-La Roche Ag | Lna oligonucleotides for splice modulation of stmn2 |
| WO2022018187A1 (en) * | 2020-07-23 | 2022-01-27 | F. Hoffmann-La Roche Ag | Oligonucleotides targeting rna binding protein sites |
| AU2022255175A1 (en) * | 2021-04-06 | 2023-11-23 | Trace Neuroscience, Inc. | Compositions and methods for treating tdp-43 proteinopathy |
| KR20240052763A (ko) * | 2021-08-11 | 2024-04-23 | 아버 바이오테크놀로지스, 인크. | 스타스민 2 (stmn2)를 표적화하는 rna 가이드를 포함하는 유전자 편집 시스템 및 이의 용도 |
| AU2022402929A1 (en) * | 2021-12-03 | 2024-06-13 | Quralis Corporation | Splice switcher antisense oligonucleotides with modified backbone chemistries |
| GB202208387D0 (en) * | 2022-06-08 | 2022-07-20 | Ucl Business Ltd | Modified U7 snRNA construct |
| GB202208384D0 (en) * | 2022-06-08 | 2022-07-20 | Ucl Business Ltd | Modified U7 snRNA construct |
| WO2024124203A2 (en) * | 2022-12-09 | 2024-06-13 | Auttx, Llc | Targeted treatment of spliceopathy-induced neurological disorders |
| WO2024173645A1 (en) * | 2023-02-15 | 2024-08-22 | Arbor Biotechnologies, Inc. | Gene editing method for inhibiting aberrant splicing in stathmin 2 (stmn2) transcript |
| WO2025184347A1 (en) * | 2024-02-27 | 2025-09-04 | Emory University | Network analysis of the cerebrospinal fluid proteome of sporadic and familial forms of amyotrophic lateral sclerosis and methods of treatment of the same |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6187545B1 (en) * | 2000-01-19 | 2001-02-13 | Isis Pharmaceuticals Inc. | Antisense modulation of pepck-cytosolic expression |
| AU2002224787A1 (en) * | 2000-10-23 | 2002-05-06 | Mermaid Pharmaceuticals Gmbh | Method for temporally controlling antisense-mediated gene inactivation |
| US20030104410A1 (en) * | 2001-03-16 | 2003-06-05 | Affymetrix, Inc. | Human microarray |
| JP2007104971A (ja) * | 2005-10-14 | 2007-04-26 | New Industry Research Organization | ミオスタチンのスプライシング制御アンチセンスオリゴヌクレオチド |
| EP1815863A1 (en) * | 2006-02-03 | 2007-08-08 | Nederlandse Organisatie voor toegepast-natuurwetenschappelijk Onderzoek TNO | Use of TLR3 agonists for the treatment of neurodegenerative disorders |
| US20110286997A1 (en) * | 2007-03-13 | 2011-11-24 | Hakon Hakonarson | Genetic Alterations on Chromosome 16 and Methods of Use Thereof for the Diagnosis and Treatment of Type 1 Diabetes |
| CA2762987A1 (en) * | 2009-05-22 | 2010-11-25 | Joseph Collard | Treatment of transcription factor e3 (tfe3) and insulin receptor substrate 2 (irs2) related diseases by inhibition of natural antisense transcript to tfe3 |
| WO2012005898A2 (en) * | 2010-06-15 | 2012-01-12 | Alnylam Pharmaceuticals, Inc. | Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof |
| WO2012177639A2 (en) * | 2011-06-22 | 2012-12-27 | Alnylam Pharmaceuticals, Inc. | Bioprocessing and bioproduction using avian cell lines |
| EP2788487B1 (en) * | 2011-12-08 | 2018-04-04 | Sarepta Therapeutics, Inc. | Oligonucleotide analogues targeting human lmna |
| ES2807379T3 (es) * | 2013-03-14 | 2021-02-22 | Ionis Pharmaceuticals Inc | Composiciones y métodos para regular la expresión de Tau |
| US20160235843A1 (en) * | 2013-10-03 | 2016-08-18 | The General Hospital Corporation | Methods for increasing neuronal survival |
| WO2016060919A1 (en) * | 2014-10-14 | 2016-04-21 | The Board Of Regents Of The University Of Texas System | Allele selective inhibition of mutant c9orf72 foci expression by duplex rnas targeting the expanded hexanucleotide repeat |
| WO2017079352A2 (en) * | 2015-11-04 | 2017-05-11 | Idera Pharmaceuticals, Inc. | Compositions for inhibiting nlrp3 gene expression and uses thereof |
| CA3103429A1 (en) * | 2018-06-14 | 2019-12-19 | Don W. Cleveland | Compounds and methods for increasing stmn2 expression |
| EP4127172A4 (en) * | 2020-03-25 | 2025-06-04 | President and Fellows of Harvard College | Methods and compositions for restoring STMN2 levels |
-
2020
- 2020-06-03 JP JP2021571911A patent/JP7795914B2/ja active Active
- 2020-06-03 WO PCT/US2020/035811 patent/WO2020247419A2/en not_active Ceased
- 2020-06-03 US US17/616,350 patent/US20220333105A1/en active Pending
- 2020-06-03 EP EP20818770.8A patent/EP3976010A4/en active Pending
- 2020-06-03 MX MX2021014868A patent/MX2021014868A/es unknown
- 2020-06-03 AU AU2020288555A patent/AU2020288555A1/en active Pending
- 2020-06-03 CA CA3142526A patent/CA3142526A1/en active Pending
- 2020-06-03 BR BR112021024463A patent/BR112021024463A2/pt unknown
- 2020-06-03 CN CN202080054507.9A patent/CN114555069A/zh active Pending
- 2020-06-03 PH PH1/2021/553024A patent/PH12021553024A1/en unknown
- 2020-06-03 KR KR1020217043411A patent/KR20220033472A/ko active Pending
-
2021
- 2021-12-01 IL IL288574A patent/IL288574A/en unknown
-
2025
- 2025-08-08 JP JP2025133172A patent/JP2026002847A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP7795914B2 (ja) | 2026-01-08 |
| EP3976010A2 (en) | 2022-04-06 |
| US20220333105A1 (en) | 2022-10-20 |
| WO2020247419A2 (en) | 2020-12-10 |
| KR20220033472A (ko) | 2022-03-16 |
| CN114555069A (zh) | 2022-05-27 |
| JP2022536085A (ja) | 2022-08-12 |
| BR112021024463A2 (pt) | 2022-03-08 |
| EP3976010A4 (en) | 2023-08-02 |
| PH12021553024A1 (en) | 2023-08-23 |
| CA3142526A1 (en) | 2020-12-10 |
| WO2020247419A3 (en) | 2021-01-14 |
| JP2026002847A (ja) | 2026-01-08 |
| AU2020288555A1 (en) | 2022-01-20 |
| IL288574A (en) | 2022-02-01 |
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