HUE057272T2 - Vírusrészecskék fokozott bevitele a csíkolt testbe és az agykéregbe - Google Patents
Vírusrészecskék fokozott bevitele a csíkolt testbe és az agykéregbeInfo
- Publication number
- HUE057272T2 HUE057272T2 HUE16714066A HUE16714066A HUE057272T2 HU E057272 T2 HUE057272 T2 HU E057272T2 HU E16714066 A HUE16714066 A HU E16714066A HU E16714066 A HUE16714066 A HU E16714066A HU E057272 T2 HUE057272 T2 HU E057272T2
- Authority
- HU
- Hungary
- Prior art keywords
- virus particles
- cerebral cortex
- increased uptake
- striped body
- striped
- Prior art date
Links
Classifications
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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
-
- 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
- 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
-
- 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
-
- 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
- A61P25/08—Antiepileptics; Anticonvulsants
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
-
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Biotechnology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Psychology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Immunology (AREA)
- Pain & Pain Management (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (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 |
|---|---|---|---|
| US201562114544P | 2015-02-10 | 2015-02-10 | |
| US201562220997P | 2015-09-19 | 2015-09-19 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HUE057272T2 true HUE057272T2 (hu) | 2022-04-28 |
Family
ID=56614902
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE16714066A HUE057272T2 (hu) | 2015-02-10 | 2016-02-09 | Vírusrészecskék fokozott bevitele a csíkolt testbe és az agykéregbe |
Country Status (27)
| Country | Link |
|---|---|
| US (3) | US20190111157A1 (https=) |
| EP (3) | EP4023758A1 (https=) |
| JP (4) | JP6928558B2 (https=) |
| KR (2) | KR102625784B1 (https=) |
| CN (2) | CN116019934A (https=) |
| AU (3) | AU2016219398A1 (https=) |
| BR (1) | BR112017017037A2 (https=) |
| CA (1) | CA2976082A1 (https=) |
| CL (1) | CL2017002028A1 (https=) |
| CR (1) | CR20170407A (https=) |
| DK (1) | DK3256594T3 (https=) |
| EA (1) | EA201791803A1 (https=) |
| ES (1) | ES2904504T3 (https=) |
| HU (1) | HUE057272T2 (https=) |
| IL (4) | IL313840A (https=) |
| LT (1) | LT3256594T (https=) |
| MX (2) | MX388264B (https=) |
| NZ (1) | NZ772772A (https=) |
| PH (1) | PH12017501436A1 (https=) |
| PL (1) | PL3256594T3 (https=) |
| PT (1) | PT3256594T (https=) |
| RS (1) | RS62811B1 (https=) |
| SG (3) | SG10201907399RA (https=) |
| SI (1) | SI3256594T1 (https=) |
| TN (1) | TN2017000353A1 (https=) |
| TW (1) | TWI730952B (https=) |
| WO (1) | WO2016130591A2 (https=) |
Families Citing this family (48)
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| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| SG11201703148TA (en) | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| CA3193811A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN119876138A (zh) | 2014-11-14 | 2025-04-25 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| HK1245326A1 (zh) | 2014-12-12 | 2018-08-24 | Voyager Therapeutics, Inc. | 用於生产scaav的组合物和方法 |
| TN2017000353A1 (en) * | 2015-02-10 | 2019-01-16 | Genzyme Corp | Enhanced delivery of viral particles to the striatum and cortex |
| TWI707951B (zh) | 2015-04-08 | 2020-10-21 | 美商健臻公司 | 過大腺相關載體之製造 |
| CA3019315A1 (en) | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| RU2742352C2 (ru) | 2015-09-24 | 2021-02-05 | Байомарин Фармасьютикал Инк. | Векторы фактора fviii на основе аденоассоциированных вирусов, соответствующие вирусные частицы и содержащие их терапевтические композиции |
| EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| CA3024448C (en) | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| CA3024449A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| JP7059285B2 (ja) * | 2016-09-30 | 2022-04-25 | エステベ プアルマセウティカルス， エッセ．ア． | ムコ多糖症の治療のためのアデノ随伴ウイルスベクター |
| EP3613856A4 (en) * | 2017-03-31 | 2020-12-23 | Staidson(Beijing) Biopharmaceuticals Co., Ltd. | SHORT RNA EXPRESSION CASSETTE IN HAIRPIN, POLYNUCLEOTIDIAL SEQUENCE WEARING IT AND CORRESPONDING APPLICATION |
| WO2018191450A2 (en) | 2017-04-14 | 2018-10-18 | National Taiwan University Hospital | Gene therapy for aadc deficiency |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| JP2020518259A (ja) * | 2017-05-05 | 2020-06-25 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | ハンチントン病治療組成物および方法 |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| 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 |
| US20200263199A1 (en) | 2017-09-29 | 2020-08-20 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| BR112020020836A2 (pt) | 2018-04-13 | 2021-01-19 | University Of Massachusetts | Vetores de aav bicistrônicos codificando subunidades alfa e beta de hexosaminidase e usos dos mesmos |
| SG11202011296VA (en) | 2018-05-15 | 2020-12-30 | Voyager Therapeutics Inc | Compositions and methods for the treatment of parkinson's disease |
| CN113528510A (zh) * | 2018-06-29 | 2021-10-22 | 武汉纽福斯生物科技有限公司 | 治疗遗传性视神经病变的组合物和方法 |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| US12448629B2 (en) | 2018-10-05 | 2025-10-21 | University Of Massachusetts | rAAV vectors for the treatment of GM1 and GM2 gangliosidosis |
| EP3880235A4 (en) * | 2018-11-14 | 2022-08-10 | REGENXBIO Inc. | GENE THERAPY OF NEURAL ZEROID LIPOFUSCINOSES |
| BR112021015817A2 (pt) * | 2019-02-22 | 2021-10-13 | The Trustees Of The University Of Pennsylvania | Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn |
| US20220307024A1 (en) * | 2019-06-17 | 2022-09-29 | Alnylam Pharmaceuticals, Inc. | Delivery of oligonucleotides to the striatum |
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| US20220275367A1 (en) * | 2019-07-24 | 2022-09-01 | Voyager Therapeutics, Inc. | Compositions and methods for treating huntington's disease |
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| JP2023517230A (ja) * | 2020-03-11 | 2023-04-24 | リモーター セラピューティクス インコーポレイテッド | 中枢神経系全体にわたってタンパク質を散在させるための方法および材料 |
| CN116171166A (zh) * | 2020-06-30 | 2023-05-26 | 塞其里斯英国有限公司 | 水包油乳液佐剂的冷过滤 |
| JP2024505885A (ja) * | 2021-01-27 | 2024-02-08 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | リソソーム酸リパーゼ欠損症（ｌａｌ－ｄ）の治療のための材料及び方法 |
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| WO2023113806A1 (en) | 2021-12-16 | 2023-06-22 | Affinia Therapeutics, Inc. | Recombinant aav for treatment of neural disease |
| WO2023108507A1 (en) * | 2021-12-15 | 2023-06-22 | National Institute Of Biological Sciences, Beijing | Recombinant aav vectors and use thereof |
| US20250333758A1 (en) * | 2021-12-15 | 2025-10-30 | Genans Biotechnology Co., Ltd | Microglia having car and use thereof |
| EP4499155A4 (en) * | 2022-03-25 | 2026-04-01 | Askbio Inc | METHODS AND COMPOSITIONS INTENDED FOR THE TREATMENT OF PARKINSON'S DISEASE |
| WO2024086747A1 (en) | 2022-10-19 | 2024-04-25 | Affinia Therapeutics Inc. | Recombinant aavs with improved tropism and specificity |
| WO2025151454A1 (en) * | 2024-01-08 | 2025-07-17 | AskBio Inc. | Method or system of infusing and/or predicting an infusate volume |
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| BRPI1007155A2 (pt) | 2009-01-29 | 2017-05-30 | Univ Of California San Francisco | métodos para tratar um distúrbio neurológico cortical, e para dispensar um agente terapêutico para o córtex em um primata |
| HUE028341T2 (en) | 2009-06-16 | 2016-12-28 | Genzyme Corp | Improved methods for purification of recombinant aav vectors |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| ES2739804T3 (es) * | 2011-02-12 | 2020-02-04 | Univ Iowa Res Found | Compuestos terapéuticos |
| DK2675902T3 (da) | 2011-02-17 | 2019-06-03 | Univ Pennsylvania | Sammensætninger og fremgangsmåder til at ændre vævsspecificitet og forbedre aav9-medieret genoverførsel |
| IL248102B (en) * | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| TN2017000353A1 (en) * | 2015-02-10 | 2019-01-16 | Genzyme Corp | Enhanced delivery of viral particles to the striatum and cortex |
| CN111417231A (zh) | 2019-01-04 | 2020-07-14 | 青岛海尔股份有限公司 | 电磁波发生系统及具有该电磁波发生系统的加热装置 |
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2016
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