CO2021011040A2 - Virus adenoasociado recombinante para el tratamiento de la neurodegeneración de inicio en adultos asociada a grn - Google Patents
Virus adenoasociado recombinante para el tratamiento de la neurodegeneración de inicio en adultos asociada a grnInfo
- Publication number
- CO2021011040A2 CO2021011040A2 CONC2021/0011040A CO2021011040A CO2021011040A2 CO 2021011040 A2 CO2021011040 A2 CO 2021011040A2 CO 2021011040 A CO2021011040 A CO 2021011040A CO 2021011040 A2 CO2021011040 A2 CO 2021011040A2
- Authority
- CO
- Colombia
- Prior art keywords
- progranulin
- aav
- grn
- associated virus
- capsid
- Prior art date
Links
- 241000702421 Dependoparvovirus Species 0.000 title abstract 7
- 230000004770 neurodegeneration Effects 0.000 title abstract 3
- 210000000234 capsid Anatomy 0.000 abstract 4
- 201000011240 Frontotemporal dementia Diseases 0.000 abstract 3
- 102000019204 Progranulins Human genes 0.000 abstract 3
- 108010012809 Progranulins Proteins 0.000 abstract 3
- 241001655883 Adeno-associated virus - 1 Species 0.000 abstract 2
- 108091026890 Coding region Proteins 0.000 abstract 2
- 108060003393 Granulin Proteins 0.000 abstract 2
- 101001027324 Homo sapiens Progranulin Proteins 0.000 abstract 2
- 210000003169 central nervous system Anatomy 0.000 abstract 2
- 102000017941 granulin Human genes 0.000 abstract 2
- 102000054121 human GRN Human genes 0.000 abstract 2
- YIEDSISPYKQADU-UHFFFAOYSA-N n-acetyl-n-[2-methyl-4-[(2-methylphenyl)diazenyl]phenyl]acetamide Chemical compound C1=C(C)C(N(C(C)=O)C(=O)C)=CC=C1N=NC1=CC=CC=C1C YIEDSISPYKQADU-UHFFFAOYSA-N 0.000 abstract 2
- 230000001105 regulatory effect Effects 0.000 abstract 2
- 102100037632 Progranulin Human genes 0.000 abstract 1
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
- 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
- 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
- C07K14/46—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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- C12N2750/14171—Demonstrated in vivo effect
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychology (AREA)
- Immunology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962809329P | 2019-02-22 | 2019-02-22 | |
| US201962923812P | 2019-10-21 | 2019-10-21 | |
| US202062969108P | 2020-02-02 | 2020-02-02 | |
| PCT/US2020/019149 WO2020172490A1 (en) | 2019-02-22 | 2020-02-21 | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2021011040A2 true CO2021011040A2 (es) | 2021-09-09 |
Family
ID=70058449
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2021/0011040A CO2021011040A2 (es) | 2019-02-22 | 2021-08-23 | Virus adenoasociado recombinante para el tratamiento de la neurodegeneración de inicio en adultos asociada a grn |
Country Status (16)
| Country | Link |
|---|---|
| US (2) | US12416017B2 (https=) |
| EP (1) | EP3927381A1 (https=) |
| JP (2) | JP7637058B2 (https=) |
| KR (1) | KR20210131370A (https=) |
| CN (1) | CN113710281A (https=) |
| AU (1) | AU2020225472A1 (https=) |
| BR (1) | BR112021015817A2 (https=) |
| CA (1) | CA3129672A1 (https=) |
| CL (1) | CL2021002172A1 (https=) |
| CO (1) | CO2021011040A2 (https=) |
| IL (1) | IL285654A (https=) |
| MX (1) | MX2021010134A (https=) |
| PE (1) | PE20211819A1 (https=) |
| SG (1) | SG11202108504YA (https=) |
| TW (1) | TW202045730A (https=) |
| WO (1) | WO2020172490A1 (https=) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220111005A1 (en) * | 2019-02-01 | 2022-04-14 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| 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 |
| GB201913974D0 (en) * | 2019-09-27 | 2019-11-13 | King S College London | Vector |
| IL292382A (en) * | 2019-10-22 | 2022-06-01 | Applied Genetic Tech Corporation | Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegenerative diseases or disorders |
| EP4204014A1 (en) * | 2020-08-26 | 2023-07-05 | The Trustees of The University of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| US20240033332A1 (en) * | 2020-12-17 | 2024-02-01 | Kyoto University | Agent for activating a neuron |
| EP4473123A4 (en) * | 2022-02-01 | 2026-03-11 | Shape Therapeutics Inc | STABLE CELL LINES FOR INDUCIBLE VAAR VIRION PRODUCTION |
| WO2024245120A1 (en) * | 2023-05-26 | 2024-12-05 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant aav vectors for treating proteinopathies in central nervous system |
| WO2024263567A2 (en) * | 2023-06-21 | 2024-12-26 | Eli Lilly And Company | Compounds for the delivery of granulin across the blood brain barrier |
| TW202548022A (zh) * | 2024-03-03 | 2025-12-16 | 美商帕西奇生物公司 | 用於治療神經退化性病症之重組腺相關病毒 |
Family Cites Families (46)
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| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| WO1998046728A1 (en) | 1997-04-14 | 1998-10-22 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
| WO1999061643A1 (en) | 1998-05-27 | 1999-12-02 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient |
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| CA2349838C (en) | 1998-11-05 | 2011-06-07 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| CA2653974A1 (en) | 2006-05-30 | 2008-02-14 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
| WO2007146046A2 (en) | 2006-06-07 | 2007-12-21 | Genzyme Corporation | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
| WO2009089635A1 (en) | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
| JP5879256B2 (ja) | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
| US20110203007A1 (en) | 2010-02-16 | 2011-08-18 | Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College | Assays of neurodegenerative disorders, including frontotemporal dementia and amyotrophic lateral sclerosis |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US20150352185A1 (en) | 2010-11-16 | 2015-12-10 | Denis G. Kay | Method for increasing neprilysin expression and activity |
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| WO2015164723A1 (en) * | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
| TN2017000353A1 (en) * | 2015-02-10 | 2019-01-16 | Genzyme Corp | Enhanced delivery of viral particles to the striatum and cortex |
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| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
| US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| EP3411488A1 (en) | 2016-02-03 | 2018-12-12 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| WO2017151884A1 (en) * | 2016-03-02 | 2017-09-08 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| US20210284701A1 (en) | 2016-07-14 | 2021-09-16 | Emory University | Granulin Compositions and Uses Related Thereto |
| WO2018045347A1 (en) | 2016-09-02 | 2018-03-08 | Spark Therapeutics, Inc. | Methods and vectors for treating cns disorders |
| JP7455579B2 (ja) | 2017-02-28 | 2024-03-26 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 新規アデノ随伴ウイルス（ａａｖ）クレードｆベクター及びその用途 |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| BR112020006671A2 (pt) | 2017-10-03 | 2020-12-01 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| US20200283800A1 (en) | 2017-10-23 | 2020-09-10 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| US20220111005A1 (en) | 2019-02-01 | 2022-04-14 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| 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 |
| EP4204014A1 (en) | 2020-08-26 | 2023-07-05 | The Trustees of The University of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
-
2020
- 2020-02-21 BR BR112021015817-0A patent/BR112021015817A2/pt unknown
- 2020-02-21 TW TW109105680A patent/TW202045730A/zh unknown
- 2020-02-21 KR KR1020217029575A patent/KR20210131370A/ko active Pending
- 2020-02-21 CA CA3129672A patent/CA3129672A1/en active Pending
- 2020-02-21 PE PE2021001357A patent/PE20211819A1/es unknown
- 2020-02-21 AU AU2020225472A patent/AU2020225472A1/en active Pending
- 2020-02-21 CN CN202080015962.8A patent/CN113710281A/zh active Pending
- 2020-02-21 US US17/431,937 patent/US12416017B2/en active Active
- 2020-02-21 MX MX2021010134A patent/MX2021010134A/es unknown
- 2020-02-21 SG SG11202108504YA patent/SG11202108504YA/en unknown
- 2020-02-21 EP EP20715985.6A patent/EP3927381A1/en active Pending
- 2020-02-21 JP JP2021549291A patent/JP7637058B2/ja active Active
- 2020-02-21 WO PCT/US2020/019149 patent/WO2020172490A1/en not_active Ceased
-
2021
- 2021-08-16 IL IL285654A patent/IL285654A/en unknown
- 2021-08-16 CL CL2021002172A patent/CL2021002172A1/es unknown
- 2021-08-23 CO CONC2021/0011040A patent/CO2021011040A2/es unknown
-
2025
- 2025-02-14 JP JP2025022078A patent/JP2025081439A/ja active Pending
- 2025-08-22 US US19/307,613 patent/US20260049335A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3927381A1 (en) | 2021-12-29 |
| BR112021015817A2 (pt) | 2021-10-13 |
| AU2020225472A1 (en) | 2021-08-26 |
| PE20211819A1 (es) | 2021-09-14 |
| US12416017B2 (en) | 2025-09-16 |
| JP2022523766A (ja) | 2022-04-26 |
| MX2021010134A (es) | 2021-09-23 |
| CL2021002172A1 (es) | 2022-02-11 |
| CA3129672A1 (en) | 2020-08-27 |
| CN113710281A (zh) | 2021-11-26 |
| US20220136008A1 (en) | 2022-05-05 |
| IL285654A (en) | 2021-09-30 |
| JP2025081439A (ja) | 2025-05-27 |
| TW202045730A (zh) | 2020-12-16 |
| KR20210131370A (ko) | 2021-11-02 |
| WO2020172490A1 (en) | 2020-08-27 |
| JP7637058B2 (ja) | 2025-02-27 |
| SG11202108504YA (en) | 2021-09-29 |
| US20260049335A1 (en) | 2026-02-19 |
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