MX2021006253A - Terapias génicas para enfermedad neurodegenerativa. - Google Patents
Terapias génicas para enfermedad neurodegenerativa.Info
- Publication number
- MX2021006253A MX2021006253A MX2021006253A MX2021006253A MX2021006253A MX 2021006253 A MX2021006253 A MX 2021006253A MX 2021006253 A MX2021006253 A MX 2021006253A MX 2021006253 A MX2021006253 A MX 2021006253A MX 2021006253 A MX2021006253 A MX 2021006253A
- Authority
- MX
- Mexico
- Prior art keywords
- disease
- neurodegenerative disease
- gene therapies
- disclosure provides
- methods
- Prior art date
Links
- 230000004770 neurodegeneration Effects 0.000 title abstract 2
- 208000015122 neurodegenerative disease Diseases 0.000 title abstract 2
- 238000001415 gene therapy Methods 0.000 title 1
- 101150037123 APOE gene Proteins 0.000 abstract 2
- 208000024827 Alzheimer disease Diseases 0.000 abstract 2
- 102100029470 Apolipoprotein E Human genes 0.000 abstract 1
- 108010029485 Protein Isoforms Proteins 0.000 abstract 1
- 102000001708 Protein Isoforms Human genes 0.000 abstract 1
- 108700019146 Transgenes Proteins 0.000 abstract 1
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
Classifications
-
- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/775—Apolipopeptides
-
- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene 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
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Hospice & Palliative Care (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Psychiatry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862772230P | 2018-11-28 | 2018-11-28 | |
| PCT/US2019/063289 WO2020112802A1 (en) | 2018-11-28 | 2019-11-26 | Gene therapies for neurodegenerative disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021006253A true MX2021006253A (es) | 2021-09-21 |
Family
ID=70852200
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021006253A MX2021006253A (es) | 2018-11-28 | 2019-11-26 | Terapias génicas para enfermedad neurodegenerativa. |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20220010001A1 (https=) |
| EP (1) | EP3887396A4 (https=) |
| JP (2) | JP7616995B2 (https=) |
| KR (1) | KR20210096168A (https=) |
| CN (1) | CN113557243A (https=) |
| AU (1) | AU2019388975A1 (https=) |
| BR (1) | BR112021010234A2 (https=) |
| CA (1) | CA3121211A1 (https=) |
| IL (1) | IL283496B2 (https=) |
| MX (1) | MX2021006253A (https=) |
| WO (1) | WO2020112802A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112020006671A2 (pt) | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| EP4045653A1 (en) * | 2019-10-16 | 2022-08-24 | Cornell University | Gene therapy for alzheimer's disease |
| EP4232572A4 (en) * | 2020-10-22 | 2024-09-25 | Duke University | COMPOSITIONS AND PROCEDURES RELATED TO ALZHEIMER’S DISEASE |
| KR20230112672A (ko) * | 2020-11-25 | 2023-07-27 | 프리베일 테라퓨틱스, 인크. | 신경변성 질환을 위한 유전자 요법 |
| EP4426833A1 (en) * | 2021-11-03 | 2024-09-11 | F. Hoffmann-La Roche AG | Oligonucleotides for modulating apolipoprotein e4 expression |
| EP4615981A1 (en) | 2022-11-11 | 2025-09-17 | Eli Lilly and Company | Synthetic nucleic acids including astrocyte-directed promoter constructs and methods of using the same |
| CN119220609B (zh) * | 2024-09-27 | 2025-09-12 | 科辉智药(深圳)新药研究中心有限公司 | 一种共表达TRIM11和ApoE2的核酸序列构建体及其应用 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP3265577B2 (ja) * | 1992-10-13 | 2002-03-11 | デューク・ユニバーシティ | アポリポ蛋白ｅの４型イソ型の測定法 |
| AU2001253233B2 (en) * | 2000-04-06 | 2006-12-07 | Kos Pharmaceuticals, Inc. | Compounds and methods for lowering cholesterol levels without inducing hypertriglyceridemia |
| US20050019927A1 (en) * | 2003-07-13 | 2005-01-27 | Markus Hildinger | DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER |
| US8435539B2 (en) * | 2004-02-13 | 2013-05-07 | Immunomedics, Inc. | Delivery system for cytotoxic drugs by bispecific antibody pretargeting |
| CA2643048A1 (en) * | 2006-02-21 | 2007-08-30 | Oklahoma Medical Research Foundation | Treatment of alzheimer's disease with inhibitors of apoe binding to apoe receptor |
| JP6469000B2 (ja) * | 2012-05-18 | 2019-02-13 | ユニバーシティー オブ アイオワ リサーチ ファウンデーション | アミロイドの沈着を処置するための方法および組成物 |
| WO2013181618A2 (en) * | 2012-05-31 | 2013-12-05 | The Trustees Of Columbia University In The City Of New York | Methods to treat alzheimer's disease using apoe inhibitors |
| EP3626309B1 (en) * | 2012-08-13 | 2023-03-08 | The Rockefeller University | Lxrbeta agonist for the treatment of cancer |
| MX2016006584A (es) * | 2013-11-20 | 2016-09-06 | Univ Iowa Res Found | Métodos y composiciones para tratar los depósitos de amiloide. |
| WO2015153760A2 (en) * | 2014-04-01 | 2015-10-08 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a nervous system disorder |
| EP3842514A1 (en) * | 2014-09-12 | 2021-06-30 | Whitehead Institute for Biomedical Research | Cells expressing apolipoprotein e and uses thereof |
| WO2018045347A1 (en) * | 2016-09-02 | 2018-03-08 | Spark Therapeutics, Inc. | Methods and vectors for treating cns disorders |
| KR102719222B1 (ko) * | 2017-05-11 | 2024-10-21 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 신경 세로이드 지질갈색소증에 대한 유전자 요법 |
| EP4045653A1 (en) * | 2019-10-16 | 2022-08-24 | Cornell University | Gene therapy for alzheimer's disease |
-
2019
- 2019-11-26 JP JP2021530800A patent/JP7616995B2/ja active Active
- 2019-11-26 MX MX2021006253A patent/MX2021006253A/es unknown
- 2019-11-26 CA CA3121211A patent/CA3121211A1/en active Pending
- 2019-11-26 US US17/297,492 patent/US20220010001A1/en not_active Abandoned
- 2019-11-26 AU AU2019388975A patent/AU2019388975A1/en active Pending
- 2019-11-26 EP EP19889757.1A patent/EP3887396A4/en active Pending
- 2019-11-26 CN CN201980086952.0A patent/CN113557243A/zh active Pending
- 2019-11-26 BR BR112021010234-5A patent/BR112021010234A2/pt unknown
- 2019-11-26 IL IL283496A patent/IL283496B2/en unknown
- 2019-11-26 KR KR1020217019516A patent/KR20210096168A/ko not_active Ceased
- 2019-11-26 WO PCT/US2019/063289 patent/WO2020112802A1/en not_active Ceased
-
2024
- 2024-08-05 JP JP2024128634A patent/JP2024177670A/ja not_active Withdrawn
Also Published As
| Publication number | Publication date |
|---|---|
| US20220010001A1 (en) | 2022-01-13 |
| CA3121211A1 (en) | 2020-06-04 |
| IL283496B1 (en) | 2025-05-01 |
| IL283496B2 (en) | 2025-09-01 |
| JP7616995B2 (ja) | 2025-01-17 |
| JP2024177670A (ja) | 2024-12-20 |
| KR20210096168A (ko) | 2021-08-04 |
| BR112021010234A2 (pt) | 2021-08-24 |
| AU2019388975A1 (en) | 2021-06-24 |
| EP3887396A4 (en) | 2022-09-07 |
| IL283496A (en) | 2021-07-29 |
| JP2022511453A (ja) | 2022-01-31 |
| CN113557243A (zh) | 2021-10-26 |
| WO2020112802A1 (en) | 2020-06-04 |
| EP3887396A1 (en) | 2021-10-06 |
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