BR112022007010A2 - Terapia gênica para doença de alzheimer - Google Patents
Terapia gênica para doença de alzheimerInfo
- Publication number
- BR112022007010A2 BR112022007010A2 BR112022007010A BR112022007010A BR112022007010A2 BR 112022007010 A2 BR112022007010 A2 BR 112022007010A2 BR 112022007010 A BR112022007010 A BR 112022007010A BR 112022007010 A BR112022007010 A BR 112022007010A BR 112022007010 A2 BR112022007010 A2 BR 112022007010A2
- Authority
- BR
- Brazil
- Prior art keywords
- disease
- alzheimer
- gene therapy
- apoe4
- mammal
- Prior art date
Links
- 208000024827 Alzheimer disease Diseases 0.000 title abstract 2
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 241000124008 Mammalia Species 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 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
- C12N15/63—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
- 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
-
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
- A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
- A01K2267/0312—Animal model for Alzheimer's disease
-
- 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
- C12N2310/50—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
- 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)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hospice & Palliative Care (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Psychiatry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962915988P | 2019-10-16 | 2019-10-16 | |
| PCT/US2020/056051 WO2021076941A1 (en) | 2019-10-16 | 2020-10-16 | Gene therapy for alzheimer's disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022007010A2 true BR112022007010A2 (pt) | 2022-07-12 |
Family
ID=73288693
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022007010A BR112022007010A2 (pt) | 2019-10-16 | 2020-10-16 | Terapia gênica para doença de alzheimer |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20230405148A1 (https=) |
| EP (1) | EP4045653A1 (https=) |
| JP (2) | JP2023500793A (https=) |
| KR (1) | KR20220082050A (https=) |
| CN (1) | CN114761569A (https=) |
| AU (2) | AU2020367437B2 (https=) |
| BR (1) | BR112022007010A2 (https=) |
| CA (1) | CA3157864A1 (https=) |
| CO (1) | CO2022006367A2 (https=) |
| IL (1) | IL292148A (https=) |
| MX (1) | MX2022004524A (https=) |
| WO (1) | WO2021076941A1 (https=) |
| ZA (1) | ZA202205344B (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7616995B2 (ja) * | 2018-11-28 | 2025-01-17 | プリベイル セラピューティクス，インコーポレーテッド | 神経変性疾患のための遺伝子治療 |
| EP4065600A1 (en) | 2019-11-25 | 2022-10-05 | Cornell University | Apoe gene therapy |
| JP2025513066A (ja) * | 2022-04-12 | 2025-04-22 | ユニキュアー バイオファーマ ビー．ブイ． | Ａｐｏｅの核酸制御 |
| EP4551711A1 (en) * | 2022-07-08 | 2025-05-14 | Cornell University | Methods and pharmaceutical compositions for the treatment and the prevention of alzheimers disease |
| WO2025213111A2 (en) * | 2024-04-05 | 2025-10-09 | Cornell University | Aav expression of mirna to suppress human apoe mrna |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US4863457A (en) | 1986-11-24 | 1989-09-05 | Lee David A | Drug delivery device |
| US5378475A (en) | 1991-02-21 | 1995-01-03 | University Of Kentucky Research Foundation | Sustained release drug delivery devices |
| AU4198793A (en) | 1992-07-24 | 1994-01-27 | Takeda Chemical Industries Ltd. | Microparticle preparation and production thereof |
| FR2695563B1 (fr) | 1992-09-11 | 1994-12-02 | Pasteur Institut | Microparticules portant des antigènes et leur utilisation pour l'induction de réponses humorales ou cellulaires. |
| ES2113094T3 (es) | 1993-03-09 | 1998-04-16 | Epic Therapeutics Inc | Las microparticulas macromoleculares y metodos de obtencion. |
| US5981719A (en) | 1993-03-09 | 1999-11-09 | Epic Therapeutics, Inc. | Macromolecular microparticles and methods of production and use |
| US6090925A (en) | 1993-03-09 | 2000-07-18 | Epic Therapeutics, Inc. | Macromolecular microparticles and methods of production and use |
| US5603960A (en) | 1993-05-25 | 1997-02-18 | O'hagan; Derek T. | Preparation of microparticles and method of immunization |
| US5464758A (en) | 1993-06-14 | 1995-11-07 | Gossen; Manfred | Tight control of gene expression in eucaryotic cells by tetracycline-responsive promoters |
| US5814618A (en) | 1993-06-14 | 1998-09-29 | Basf Aktiengesellschaft | Methods for regulating gene expression |
| US6913767B1 (en) | 1993-10-25 | 2005-07-05 | Genentech, Inc. | Compositions for microencapsulation of antigens for use as vaccines |
| US5443505A (en) | 1993-11-15 | 1995-08-22 | Oculex Pharmaceuticals, Inc. | Biocompatible ocular implants |
| US6342390B1 (en) | 1994-11-23 | 2002-01-29 | The United States Of America As Represented By The Secretary Of Health And Human Services | Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy |
| EP0930874A2 (en) | 1996-10-09 | 1999-07-28 | Takeda Chemical Industries, Ltd. | A method for producing a microparticle |
| US6210707B1 (en) | 1996-11-12 | 2001-04-03 | The Regents Of The University Of California | Methods of forming protein-linked lipidic microparticles, and compositions thereof |
| US6884435B1 (en) | 1997-01-30 | 2005-04-26 | Chiron Corporation | Microparticles with adsorbent surfaces, methods of making same, and uses thereof |
| US6406719B1 (en) | 1998-05-13 | 2002-06-18 | Microbiological Research Authority | Encapsulation of bioactive agents |
| GB9810236D0 (en) | 1998-05-13 | 1998-07-08 | Microbiological Res Authority | Improvements relating to encapsulation of bioactive agents |
| US6264987B1 (en) | 2000-05-19 | 2001-07-24 | Alkermes Controlled Therapeutics Inc. Ii | Method for preparing microparticles having a selected polymer molecular weight |
| FR2814642B1 (fr) | 2000-10-03 | 2005-07-01 | Ass Pour Le Dev De La Rech En | Souris transgenique pour la recombinaison ciblee mediee par la cre-er modifiee |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| WO2005116204A1 (ja) * | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法 |
| US20070081972A1 (en) | 2005-09-30 | 2007-04-12 | The University Of Iowa Research Foundation | Polymer-based delivery system for immunotherapy of cancer |
| US20070248599A1 (en) * | 2006-02-21 | 2007-10-25 | Jordan Tang | Treatment of alzheimer's disease with inhibitors of apoe binding to apoe receptor |
| EP2114153B1 (en) * | 2007-02-06 | 2016-11-23 | Tai June Yoo | Treatment and prevention of neurodegenerative diseases using gene therapy |
| WO2012054425A2 (en) | 2010-10-18 | 2012-04-26 | University Of Iowa Research Foundation | Biodegradable particulate formulations |
| US9345776B2 (en) | 2011-02-24 | 2016-05-24 | The University Of Iowa Research Foundation | Biodegradable polymers with sulfenamide bonds for drug delivery applications |
| CN104540952A (zh) * | 2012-05-18 | 2015-04-22 | 衣阿华大学研究基金会 | 用于治疗淀粉状蛋白沉积的方法和组合物 |
| 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 |
| IL245703B (en) * | 2013-11-20 | 2022-07-01 | Univ Iowa Res Found | Methods and compositions for treating amyloid deposits |
| JP7610923B2 (ja) * | 2016-09-02 | 2025-01-09 | スパーク セラピューティクス インコーポレイテッド | Ｃｎｓ障害を治療するための方法及びベクター |
| US11071791B2 (en) * | 2018-01-26 | 2021-07-27 | Wisconsin Alumni Research Foundation | Vector for gene silencing and replacement and methods of use thereof |
| JP7616995B2 (ja) * | 2018-11-28 | 2025-01-17 | プリベイル セラピューティクス，インコーポレーテッド | 神経変性疾患のための遺伝子治療 |
-
2020
- 2020-10-16 JP JP2022522814A patent/JP2023500793A/ja active Pending
- 2020-10-16 WO PCT/US2020/056051 patent/WO2021076941A1/en not_active Ceased
- 2020-10-16 KR KR1020227016247A patent/KR20220082050A/ko active Pending
- 2020-10-16 AU AU2020367437A patent/AU2020367437B2/en active Active
- 2020-10-16 EP EP20804387.7A patent/EP4045653A1/en active Pending
- 2020-10-16 BR BR112022007010A patent/BR112022007010A2/pt unknown
- 2020-10-16 MX MX2022004524A patent/MX2022004524A/es unknown
- 2020-10-16 CN CN202080073402.8A patent/CN114761569A/zh active Pending
- 2020-10-16 CA CA3157864A patent/CA3157864A1/en active Pending
- 2020-10-16 US US17/769,255 patent/US20230405148A1/en active Pending
-
2022
- 2022-04-11 IL IL292148A patent/IL292148A/en unknown
- 2022-05-13 ZA ZA2022/05344A patent/ZA202205344B/en unknown
- 2022-05-16 CO CONC2022/0006367A patent/CO2022006367A2/es unknown
-
2025
- 2025-02-06 AU AU2025200812A patent/AU2025200812A1/en active Pending
- 2025-12-05 JP JP2025233417A patent/JP2026041903A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| ZA202205344B (en) | 2023-03-29 |
| MX2022004524A (es) | 2022-07-21 |
| WO2021076941A1 (en) | 2021-04-22 |
| IL292148A (en) | 2022-06-01 |
| CA3157864A1 (en) | 2021-04-22 |
| WO2021076941A9 (en) | 2021-10-07 |
| JP2023500793A (ja) | 2023-01-11 |
| CO2022006367A2 (es) | 2022-08-09 |
| US20230405148A1 (en) | 2023-12-21 |
| AU2020367437A1 (en) | 2022-06-02 |
| EP4045653A1 (en) | 2022-08-24 |
| KR20220082050A (ko) | 2022-06-16 |
| CN114761569A (zh) | 2022-07-15 |
| AU2025200812A1 (en) | 2025-02-27 |
| AU2020367437B2 (en) | 2024-11-07 |
| JP2026041903A (ja) | 2026-03-10 |
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