JP7587423B2 - アルツハイマー病のための遺伝子治療 - Google Patents
アルツハイマー病のための遺伝子治療 Download PDFInfo
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- JP7587423B2 JP7587423B2 JP2020565341A JP2020565341A JP7587423B2 JP 7587423 B2 JP7587423 B2 JP 7587423B2 JP 2020565341 A JP2020565341 A JP 2020565341A JP 2020565341 A JP2020565341 A JP 2020565341A JP 7587423 B2 JP7587423 B2 JP 7587423B2
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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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6478—Aspartic endopeptidases (3.4.23)
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- 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
- C07K14/46—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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- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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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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- 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
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
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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
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
- A61K9/5184—Virus capsids or envelopes enclosing drugs
-
- 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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- 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
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/23—Aspartic endopeptidases (3.4.23)
- C12Y304/23045—Memapsin 1 (3.4.23.45), i.e. beta-secretase 2 or BACE2
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/23—Aspartic endopeptidases (3.4.23)
- C12Y304/23046—Memapsin 2 (3.4.23.46), i.e. beta-secretase 1 or BACE
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
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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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
- C12N2710/16132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
-
- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
- C12N2710/16141—Use of virus, viral particle or viral elements as a vector
- C12N2710/16143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Neurology (AREA)
- Wood Science & Technology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Neurosurgery (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Hospice & Palliative Care (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Psychiatry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Psychology (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Plant Pathology (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862675003P | 2018-05-22 | 2018-05-22 | |
| US62/675,003 | 2018-05-22 | ||
| PCT/US2019/033616 WO2019226832A1 (en) | 2018-05-22 | 2019-05-22 | Gene therapy for alzheimer's disease |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2021525245A JP2021525245A (ja) | 2021-09-24 |
| JPWO2019226832A5 JPWO2019226832A5 (https=) | 2022-05-26 |
| JP2021525245A5 JP2021525245A5 (https=) | 2022-05-26 |
| JP7587423B2 true JP7587423B2 (ja) | 2024-11-20 |
Family
ID=68617210
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020565341A Active JP7587423B2 (ja) | 2018-05-22 | 2019-05-22 | アルツハイマー病のための遺伝子治療 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US12497608B2 (https=) |
| EP (1) | EP3796980A4 (https=) |
| JP (1) | JP7587423B2 (https=) |
| CN (1) | CN112739419A (https=) |
| AU (1) | AU2019272848A1 (https=) |
| CA (1) | CA3100946A1 (https=) |
| IL (1) | IL278813A (https=) |
| WO (1) | WO2019226832A1 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3796980A4 (en) | 2018-05-22 | 2022-03-23 | The Brigham & Women's Hospital, Inc. | NEW THERAPY FOR ALZHEIMER'S DISEASE |
| WO2020242892A1 (en) | 2019-05-24 | 2020-12-03 | The Brigham And Women's Hospital, Inc. | Gene therapy for alzheimer's disease |
| IL293285A (en) * | 2019-11-29 | 2022-07-01 | Paros Bio Inc | Gene therapy for neurodegenerative disorders |
| WO2022261413A1 (en) * | 2021-06-10 | 2022-12-15 | The Brigham And Women’S Hospital, Inc. | Cell-based assay for identification of activators of gamma-secretase |
| EP4399311A4 (en) * | 2021-09-10 | 2025-11-26 | Alnylam Pharmaceuticals Inc | APP ARNI COMPOSITIONS AND THEIR METHODS OF USE FOR TREATMENT OR PREVENTION OF DISEASES CHARACTERIZED BY ENLARGED ENDOSOMES |
| CN116024270B (zh) * | 2021-12-27 | 2025-07-22 | 中国科学院昆明动物研究所 | 一种app/psen1双转基因阿尔茨海默病树鼩模型的建立方法 |
| CN115125244A (zh) * | 2022-05-14 | 2022-09-30 | 吉林大学 | 构建阿尔兹海默症的兔模型方法 |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2013509890A (ja) | 2009-11-09 | 2013-03-21 | ジーンポッド セラピューティクス アーベー | invivoでのニューロン特異的な最適化された連続DOPA合成用の新規ウイルスベクター構築物 |
| JP2013107890A (ja) | 1995-04-28 | 2013-06-06 | Hsc Research & Development Ltd | アルツハイマー病に関連する遺伝子配列およびタンパク質、ならびにその使用 |
| JP2016523835A (ja) | 2013-05-15 | 2016-08-12 | リージェンツ オブ ザ ユニバーシティ オブ ミネソタ | 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入 |
Family Cites Families (41)
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| US5328470A (en) | 1989-03-31 | 1994-07-12 | The Regents Of The University Of Michigan | Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor |
| ES2249761T3 (es) | 1993-06-24 | 2006-04-01 | Advec Inc. | Vectores de adenovirus para terapia genica. |
| EP1637608B1 (en) | 1993-10-25 | 2009-07-22 | CANJI, Inc. | Recombinant adenoviral vector and methods of use |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| US7252989B1 (en) | 1994-04-04 | 2007-08-07 | Board Of Regents, The University Of Texas System | Adenovirus supervector system |
| CA2187626C (en) | 1994-04-13 | 2009-11-03 | Michael G. Kaplitt | Aav-mediated delivery of dna to cells of the nervous system |
| AU732508B2 (en) | 1996-01-26 | 2001-04-26 | Governing Council Of The University Of Toronto, The | Nucleic acids and proteins related to Alzheimer's disease, and uses therefor |
| AR020107A1 (es) * | 1998-07-09 | 2002-04-10 | Boehringer Ingelheim Pharma | Metodo para identificar una sustancia capaz de reducir o eliminar la actividad de la presenilinasa, las sustancias identificables con dicho metodo, el usode estas ultimas en la elaboracion de un medicamento para el tratamiento de enfermedades neurodegenerativas y las composiciones farmaceuticas que |
| AU4992099A (en) * | 1998-07-16 | 2000-02-07 | Incyte Pharmaceuticals, Inc. | Human presenilin-associated protein |
| US6979537B2 (en) | 2000-01-10 | 2005-12-27 | Scios, Inc. | Methods for identifying inhibitors of neuronal degeneration |
| US6686449B2 (en) | 2000-06-30 | 2004-02-03 | Pharmacia & Upjohn Company | Mutant presenilin 1 polypeptides |
| WO2004016733A2 (en) | 2002-08-16 | 2004-02-26 | Agensys, Inc. | Nucleic acid and corresponding protein entitled 251p5g2 useful in treatment and detection of cancer |
| US7271313B2 (en) | 2003-04-09 | 2007-09-18 | The Brigham And Women's Hospital, Inc. | Presenilin-deficient mouse model of age-dependent neurodegeneration and cognitive loss |
| CA2528963A1 (en) | 2003-06-27 | 2005-01-13 | Sirna Therapeutics, Inc. | Rna interference mediated treatment of alzheimer's disease using short interfering nucleic acid (sina) |
| WO2005037226A2 (en) | 2003-10-17 | 2005-04-28 | Georgia Tech Research Corporation | Genetically engineered enteroendocrine cells for treating glucose-related metabolic disorders |
| PL1675956T3 (pl) | 2003-10-21 | 2011-06-30 | Merck Serono Sa | Minimalna sekwencja DNA działająca jako insulator chromatyny i jej zastosowanie do wyrażania białek |
| US7498316B2 (en) | 2004-04-06 | 2009-03-03 | University Of Massachusetts | Methods and compositions for treating gain-of-function disorders using RNA interference |
| CA2566519C (en) | 2004-05-14 | 2020-04-21 | Rosetta Genomics Ltd. | Micrornas and uses thereof |
| JP2008500039A (ja) | 2004-05-26 | 2008-01-10 | ロゼッタ ジノミクス リミテッド | ウイルスmiRNAおよびウイルス関連miRNAならびにその使用 |
| US8129334B2 (en) | 2006-03-31 | 2012-03-06 | The Regents Of The University Of California | Methods and compositions for treating neurodegenerative disorders and Alzheimer'S disease and improving normal memory |
| US9085778B2 (en) | 2006-05-03 | 2015-07-21 | VL27, Inc. | Exosome transfer of nucleic acids to cells |
| WO2009042727A1 (en) | 2007-09-24 | 2009-04-02 | The Johns Hopkins University | Immediate early gene arc interacts with endocytic machinery and regulates the trafficking and function of presenilin |
| WO2012075040A2 (en) | 2010-11-30 | 2012-06-07 | Shire Human Genetic Therapies, Inc. | mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES |
| CN103816540B (zh) * | 2012-11-16 | 2018-01-02 | 中国科学院上海生命科学研究院 | 降低β‑抑制蛋白1与APH‑1蛋白的结合的物质在制备防治神经退行性疾病药物中的应用 |
| HK1222673A1 (zh) * | 2013-07-12 | 2017-07-07 | The Children's Hospital Of Philadelphia | Aav载体和用於抗aav(腺相关病毒)中和抗体的检测 |
| GB201404470D0 (en) | 2014-03-13 | 2014-04-30 | Ucl Business Plc | Therapeutic methods and materials |
| GB201413665D0 (en) * | 2014-07-03 | 2014-09-17 | Transimmune Ag And Yale University | Method for obtaining globally activated monocytes |
| US10350245B2 (en) | 2015-01-21 | 2019-07-16 | Fred Hutchinson Cancer Research Center | Point-of-care and/or portable platform for gene therapy |
| JP2018510160A (ja) * | 2015-03-20 | 2018-04-12 | ブルーバード バイオ, インコーポレイテッド | ベクター製剤 |
| WO2016209654A1 (en) * | 2015-06-22 | 2016-12-29 | The Board Of Trustees Of The Leland Stanford Junior University | Methods and devices for imaging and/or optogenetic control of light-responsive neurons |
| EP3452101A2 (en) | 2016-05-04 | 2019-03-13 | CureVac AG | Rna encoding a therapeutic protein |
| EP4316588A3 (en) | 2016-05-27 | 2024-05-15 | Transcriptx, Inc. | Treatment of primary ciliary dyskinesia with synthetic messenger rna |
| WO2018045022A1 (en) | 2016-08-30 | 2018-03-08 | University Of South Florida | Adipose derived stem cell exosomes and uses thereof |
| CA3050264A1 (en) | 2017-01-24 | 2018-08-02 | Fred Hutchinson Cancer Research Center | Systems and methods for hematopoietic cell expansion utilizing hydrogels |
| US11713470B2 (en) | 2017-03-20 | 2023-08-01 | University of Pittsburgh—of the Commonwealth System of Higher Education | Targeted gene therapies for pain and other neuro-related disorders |
| ES3039647T3 (en) | 2017-05-31 | 2025-10-23 | Arcturus Therapeutics Inc | Synthesis and structure of high potency rna therapeutics |
| JP7221275B2 (ja) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Aavを送達するための組成物および方法 |
| EP3796980A4 (en) | 2018-05-22 | 2022-03-23 | The Brigham & Women's Hospital, Inc. | NEW THERAPY FOR ALZHEIMER'S DISEASE |
| WO2020242892A1 (en) | 2019-05-24 | 2020-12-03 | The Brigham And Women's Hospital, Inc. | Gene therapy for alzheimer's disease |
| IL293285A (en) | 2019-11-29 | 2022-07-01 | Paros Bio Inc | Gene therapy for neurodegenerative disorders |
| US20230136245A1 (en) | 2020-01-31 | 2023-05-04 | Paros Bio, Inc. | Gene therapy for neurodegenerative disorders using polynucleotide silencing and replacement |
-
2019
- 2019-05-22 EP EP19808081.4A patent/EP3796980A4/en active Pending
- 2019-05-22 CN CN201980049127.3A patent/CN112739419A/zh active Pending
- 2019-05-22 WO PCT/US2019/033616 patent/WO2019226832A1/en not_active Ceased
- 2019-05-22 US US17/057,001 patent/US12497608B2/en active Active
- 2019-05-22 AU AU2019272848A patent/AU2019272848A1/en not_active Abandoned
- 2019-05-22 JP JP2020565341A patent/JP7587423B2/ja active Active
- 2019-05-22 CA CA3100946A patent/CA3100946A1/en active Pending
-
2020
- 2020-11-18 IL IL278813A patent/IL278813A/en unknown
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2013107890A (ja) | 1995-04-28 | 2013-06-06 | Hsc Research & Development Ltd | アルツハイマー病に関連する遺伝子配列およびタンパク質、ならびにその使用 |
| JP2013509890A (ja) | 2009-11-09 | 2013-03-21 | ジーンポッド セラピューティクス アーベー | invivoでのニューロン特異的な最適化された連続DOPA合成用の新規ウイルスベクター構築物 |
| JP2016523835A (ja) | 2013-05-15 | 2016-08-12 | リージェンツ オブ ザ ユニバーシティ オブ ミネソタ | 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入 |
Non-Patent Citations (6)
| Title |
|---|
| Alzheimer's & Dementia,2012,Vol.8, No.4S, Part 3,p.105 |
| 'Homo sapiens presenilin 1 (PSEN1), transcript variant 1, mRNA' DATABASE NCBI Nucleotide [online] accession:NM_000021 (Version:NM_000021.3), 07-AUG-2008, [retrieved on 2023.03.09], retrieved from the internet:,https://www.ncbi.nlm.nih.gov/nuccore/195947402?report=gb&sat=12&satkey=6105349 |
| 'Homo sapiens presenilin 1 (PSEN1), transcript variant 2, mRNA' DATABASE NCBI Nucleotide [online] accession:NM_007318 (Version:NM_007318.2), 07-AUG-2008, [retrieved on 2023.03.09], retrieved from the internet: ,https://www.ncbi.nlm.nih.gov/nuccore/195947396?sat=12&satkey=6105331 |
| 'Homo sapiens presenilin 2 (Alzheimer disease 4) (PSEN2), transcript variant 1, mRNA' DATABASE NCBI Nucleotide [online] accession:NM_000447 (Version:NM_000447.2), 17-AUG-2007, [retrieved on 2023.03.09], retrieved from the internet: ,https://www.ncbi.nlm.nih.gov/nuccore/156105678?sat=11&satkey=9636665 |
| 'Homo sapiens presenilin 2 (Alzheimer disease 4) (PSEN2), transcript variant 2, mRNA' DATABASE NCBI Nucleotide [online] accession:NM_012486 (Version:NM_012486.2), 17-AUG-2007, [retrieved on 2023.03.09], retrieved from the internet: ,https://www.ncbi.nlm.nih.gov/nuccore/156105680?sat=11&satkey=9636666 |
| J. Biol. Chem.,2006年,Vol.281, No.22,pp.15330-15336 |
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| WO2019226832A1 (en) | 2019-11-28 |
| US12497608B2 (en) | 2025-12-16 |
| JP2021525245A (ja) | 2021-09-24 |
| IL278813A (en) | 2021-01-31 |
| AU2019272848A1 (en) | 2020-12-03 |
| CA3100946A1 (en) | 2019-11-28 |
| EP3796980A4 (en) | 2022-03-23 |
| US20210108186A1 (en) | 2021-04-15 |
| EP3796980A1 (en) | 2021-03-31 |
| CN112739419A (zh) | 2021-04-30 |
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