BR112022010065A2 - Terapia de gene da apoe - Google Patents
Terapia de gene da apoeInfo
- Publication number
- BR112022010065A2 BR112022010065A2 BR112022010065A BR112022010065A BR112022010065A2 BR 112022010065 A2 BR112022010065 A2 BR 112022010065A2 BR 112022010065 A BR112022010065 A BR 112022010065A BR 112022010065 A BR112022010065 A BR 112022010065A BR 112022010065 A2 BR112022010065 A2 BR 112022010065A2
- Authority
- BR
- Brazil
- Prior art keywords
- gene therapy
- apoe gene
- apoe
- vector
- mammalian apolipoprotein
- Prior art date
Links
Classifications
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
-
- 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
- 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)
- Biotechnology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Gastroenterology & Hepatology (AREA)
- Hospice & Palliative Care (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Toxicology (AREA)
- Virology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962939999P | 2019-11-25 | 2019-11-25 | |
| PCT/US2020/070822 WO2021108809A1 (en) | 2019-11-25 | 2020-11-25 | Apoe gene therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112022010065A2 true BR112022010065A2 (pt) | 2022-09-06 |
Family
ID=73857283
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022010065A BR112022010065A2 (pt) | 2019-11-25 | 2020-11-25 | Terapia de gene da apoe |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US12611467B2 (https=) |
| EP (1) | EP4065600A1 (https=) |
| JP (2) | JP2023502515A (https=) |
| KR (1) | KR20220107243A (https=) |
| CN (1) | CN114829390A (https=) |
| AU (1) | AU2020391518B2 (https=) |
| BR (1) | BR112022010065A2 (https=) |
| CA (1) | CA3162761A1 (https=) |
| IL (1) | IL293116A (https=) |
| MX (1) | MX2022006364A (https=) |
| NZ (1) | NZ789753A (https=) |
| WO (1) | WO2021108809A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN119264211A (zh) | 2018-08-27 | 2025-01-07 | 瑞泽恩制药公司 | 拉曼光谱在下游纯化中的应用 |
| WO2020160156A2 (en) | 2019-01-30 | 2020-08-06 | Immutics, Inc. | Anti-gal3 antibodies and uses thereof |
| EP4065600A1 (en) | 2019-11-25 | 2022-10-05 | Cornell University | Apoe gene therapy |
| EP4157338A4 (en) | 2020-05-26 | 2024-11-13 | TrueBinding, Inc. | METHOD FOR TREATING INFLAMMATORY DISEASES BY GALECTIN-3 BLOCKING |
| KR20230112672A (ko) * | 2020-11-25 | 2023-07-27 | 프리베일 테라퓨틱스, 인크. | 신경변성 질환을 위한 유전자 요법 |
| WO2023198702A1 (en) * | 2022-04-12 | 2023-10-19 | Uniqure Biopharma B.V. | Nucleic acid regulation of c9orf72 |
| 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 |
| CN121335984A (zh) * | 2023-04-18 | 2026-01-13 | 康奈尔大学 | 用于治疗和预防阿尔茨海默病的方法和apoe药物组合物 |
| WO2025213111A2 (en) * | 2024-04-05 | 2025-10-09 | Cornell University | Aav expression of mirna to suppress human apoe mrna |
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| US4861719A (en) | 1986-04-25 | 1989-08-29 | Fred Hutchinson Cancer Research Center | DNA constructs for retrovirus packaging cell lines |
| US4863457A (en) | 1986-11-24 | 1989-09-05 | Lee David A | Drug delivery device |
| US5278056A (en) | 1988-02-05 | 1994-01-11 | The Trustees Of Columbia University In The City Of New York | Retroviral packaging cell lines and process of using same |
| US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
| 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. |
| DE69434860T2 (de) | 1993-02-22 | 2007-03-15 | The Rockefeller University | Herstellung von helfer-freien retroviren mit hohem titer mittels transienter transfektion |
| US6090925A (en) | 1993-03-09 | 2000-07-18 | Epic Therapeutics, Inc. | Macromolecular microparticles and methods of production and use |
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| US5603960A (en) | 1993-05-25 | 1997-02-18 | O'hagan; Derek T. | Preparation of microparticles and method of immunization |
| ATE251886T1 (de) | 1993-08-31 | 2003-11-15 | Univ Duke | Verwendunf von apoe zum binden von tau und map2c proteinen und zur behandlung von alzheimer's krankheit |
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| US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
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| 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 |
| WO2015013363A2 (en) | 2013-07-24 | 2015-01-29 | The General Hospital Corporation | Agents and methods for inhibiting mir-148a for the modulation of cholesterol levels |
| IL245703B (en) | 2013-11-20 | 2022-07-01 | Univ Iowa Res Found | Methods and compositions for treating amyloid deposits |
| CN107073051B (zh) | 2014-10-21 | 2021-08-24 | 马萨诸塞大学 | 重组aav变体及其用途 |
| EP3452074A4 (en) | 2016-05-03 | 2019-12-11 | University of South Florida | COMPOSITIONS AND METHOD FOR MODULATING THE ABETA PROTEIN |
| 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 |
| CN106890315B (zh) | 2017-03-16 | 2019-09-06 | 北京热休生物技术有限公司 | Apo-e蛋白及其多肽在治疗与预防癌症中的应用 |
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| US11680275B2 (en) | 2017-06-06 | 2023-06-20 | University Of Massachusetts | Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome |
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| US11071791B2 (en) | 2018-01-26 | 2021-07-27 | Wisconsin Alumni Research Foundation | Vector for gene silencing and replacement and methods of use thereof |
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| CN109776665B (zh) | 2019-02-02 | 2021-02-05 | 首都医科大学宣武医院 | 阿尔茨海默病新突变、其稳转细胞模型及医药用途 |
| CN113811311A (zh) | 2019-03-15 | 2021-12-17 | 马萨诸塞大学 | 用于组织特异性apoe调节的寡核苷酸 |
| AU2020282792B2 (en) | 2019-05-28 | 2025-12-04 | Banner Health | ApoE antibodies, fusion proteins and uses thereof |
| JP2023500793A (ja) | 2019-10-16 | 2023-01-11 | コーネル・ユニバーシティー | アルツハイマー病に対する遺伝子治療方法 |
| EP4065600A1 (en) | 2019-11-25 | 2022-10-05 | Cornell University | Apoe gene therapy |
| JP7539723B2 (ja) | 2020-02-21 | 2024-08-26 | コンバインド セラピューティクス インコーポレイテッド | コード化リボ核酸の器官保護発現及び調節のための組成物並びに方法 |
| CN111471716B (zh) | 2020-05-20 | 2021-10-15 | 上海市同济医院 | 一种基于重组腺相关病毒8型的microRNA-212-3P海绵的构建及其应用 |
| CN114250235A (zh) | 2020-09-21 | 2022-03-29 | 华东理工大学 | 一种重组腺相关病毒载体疫苗的设计方法及应用 |
| KR20230112672A (ko) | 2020-11-25 | 2023-07-27 | 프리베일 테라퓨틱스, 인크. | 신경변성 질환을 위한 유전자 요법 |
| EP4334446A1 (en) | 2021-05-03 | 2024-03-13 | CureVac SE | Improved nucleic acid sequence for cell type specific expression |
| 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 |
| IL316236A (en) | 2022-04-15 | 2024-12-01 | Encoded Therapeutics Inc | Elements for removing gene expression targeting in the liver |
| EP4551711A1 (en) | 2022-07-08 | 2025-05-14 | Cornell University | Methods and pharmaceutical compositions for the treatment and the prevention of alzheimers disease |
| CN116574760A (zh) | 2023-04-13 | 2023-08-11 | 中山大学肿瘤防治中心（中山大学附属肿瘤医院、中山大学肿瘤研究所） | 一种基于miR-302d骨架的高效率shRNA表达载体及其制备和应用 |
| CN121335984A (zh) | 2023-04-18 | 2026-01-13 | 康奈尔大学 | 用于治疗和预防阿尔茨海默病的方法和apoe药物组合物 |
| WO2024220722A1 (en) | 2023-04-18 | 2024-10-24 | Cornell University | Compositions and methods for tissue- specific expression of gene therapies |
| WO2025213111A2 (en) | 2024-04-05 | 2025-10-09 | Cornell University | Aav expression of mirna to suppress human apoe mrna |
-
2020
- 2020-11-25 EP EP20828741.7A patent/EP4065600A1/en active Pending
- 2020-11-25 AU AU2020391518A patent/AU2020391518B2/en active Active
- 2020-11-25 IL IL293116A patent/IL293116A/en unknown
- 2020-11-25 NZ NZ789753A patent/NZ789753A/en unknown
- 2020-11-25 WO PCT/US2020/070822 patent/WO2021108809A1/en not_active Ceased
- 2020-11-25 JP JP2022530164A patent/JP2023502515A/ja active Pending
- 2020-11-25 MX MX2022006364A patent/MX2022006364A/es unknown
- 2020-11-25 BR BR112022010065A patent/BR112022010065A2/pt unknown
- 2020-11-25 US US17/779,812 patent/US12611467B2/en active Active
- 2020-11-25 KR KR1020227021579A patent/KR20220107243A/ko active Pending
- 2020-11-25 CA CA3162761A patent/CA3162761A1/en active Pending
- 2020-11-25 CN CN202080081724.7A patent/CN114829390A/zh active Pending
-
2025
- 2025-07-25 JP JP2025124433A patent/JP2025163078A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2020391518B2 (en) | 2025-02-13 |
| CA3162761A1 (en) | 2021-06-03 |
| NZ789753A (en) | 2026-02-27 |
| CN114829390A (zh) | 2022-07-29 |
| MX2022006364A (es) | 2022-06-22 |
| EP4065600A1 (en) | 2022-10-05 |
| IL293116A (en) | 2022-07-01 |
| JP2023502515A (ja) | 2023-01-24 |
| JP2025163078A (ja) | 2025-10-28 |
| AU2020391518A1 (en) | 2022-07-14 |
| US12611467B2 (en) | 2026-04-28 |
| KR20220107243A (ko) | 2022-08-02 |
| US20230044351A1 (en) | 2023-02-09 |
| WO2021108809A1 (en) | 2021-06-03 |
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