IL303156A - Gene therapies for neurodegenerative disease - Google Patents
Gene therapies for neurodegenerative diseaseInfo
- Publication number
- IL303156A IL303156A IL303156A IL30315623A IL303156A IL 303156 A IL303156 A IL 303156A IL 303156 A IL303156 A IL 303156A IL 30315623 A IL30315623 A IL 30315623A IL 303156 A IL303156 A IL 303156A
- Authority
- IL
- Israel
- Prior art keywords
- nucleic acid
- protein
- isolated nucleic
- christchurch
- apoe
- Prior art date
Links
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Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/775—Apolipopeptides
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- 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
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- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- 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
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- 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
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- 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
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
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- C07K—PEPTIDES
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- C07—ORGANIC CHEMISTRY
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- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/53—Physical structure partially self-complementary or closed
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- 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
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- C12N2800/00—Nucleic acids vectors
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- C12N2830/00—Vector systems having a special element relevant for transcription
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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- Wood Science & Technology (AREA)
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- Microbiology (AREA)
- Plant Pathology (AREA)
- Hospice & Palliative Care (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychology (AREA)
- Toxicology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
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| US202063118060P | 2020-11-25 | 2020-11-25 | |
| PCT/US2021/060731 WO2022115535A1 (en) | 2020-11-25 | 2021-11-24 | Gene therapies for neurodegenerative disease |
Publications (1)
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| IL303156A true IL303156A (en) | 2023-07-01 |
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
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| IL303156A IL303156A (en) | 2020-11-25 | 2021-11-24 | Gene therapies for neurodegenerative disease |
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| Country | Link |
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| US (1) | US20230405149A1 (https=) |
| EP (1) | EP4251276A4 (https=) |
| JP (1) | JP2023551254A (https=) |
| KR (1) | KR20230112672A (https=) |
| CN (1) | CN116670291A (https=) |
| AU (1) | AU2021386390A1 (https=) |
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| MX (1) | MX2023006153A (https=) |
| WO (1) | WO2022115535A1 (https=) |
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| AU2024258754A1 (en) * | 2023-04-18 | 2025-11-13 | Cornell University | Methods and apoe pharmaceutical compositions for the treatment and the prevention of alzheimers disease |
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| WO2000023587A2 (en) * | 1998-10-16 | 2000-04-27 | Introgene B.V. | Gene therapy of alzheimer's disease by delivery of an encoded apolipoprotein e |
| AU2001253233B2 (en) * | 2000-04-06 | 2006-12-07 | Kos Pharmaceuticals, Inc. | Compounds and methods for lowering cholesterol levels without inducing hypertriglyceridemia |
| 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 |
| 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 |
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2021
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- 2021-11-24 EP EP21899082.8A patent/EP4251276A4/en active Pending
- 2021-11-24 AU AU2021386390A patent/AU2021386390A1/en not_active Abandoned
- 2021-11-24 WO PCT/US2021/060731 patent/WO2022115535A1/en not_active Ceased
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| CN116670291A (zh) | 2023-08-29 |
| AU2021386390A1 (en) | 2023-06-29 |
| JP2023551254A (ja) | 2023-12-07 |
| WO2022115535A1 (en) | 2022-06-02 |
| EP4251276A4 (en) | 2024-10-23 |
| CA3203006A1 (en) | 2022-06-02 |
| EP4251276A1 (en) | 2023-10-04 |
| KR20230112672A (ko) | 2023-07-27 |
| US20230405149A1 (en) | 2023-12-21 |
| MX2023006153A (es) | 2023-07-18 |
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