MX2021010134A - Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn. - Google Patents
Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn.Info
- Publication number
- MX2021010134A MX2021010134A MX2021010134A MX2021010134A MX2021010134A MX 2021010134 A MX2021010134 A MX 2021010134A MX 2021010134 A MX2021010134 A MX 2021010134A MX 2021010134 A MX2021010134 A MX 2021010134A MX 2021010134 A MX2021010134 A MX 2021010134A
- Authority
- MX
- Mexico
- Prior art keywords
- progranulin
- grn
- associated virus
- raav
- aav
- Prior art date
Links
Classifications
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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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- 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
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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
- 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
- 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
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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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
- C12N2750/14141—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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- 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)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- General Chemical & Material Sciences (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychology (AREA)
- Immunology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
Se proporciona un AAV recombinante (rAAV) adecuado para su uso en el tratamiento de la neurodegeneración de inicio en adultos causada por la haploinsuficiencia de granulina (GRN), tal como la demencia frontotemporal (FTD) relacionada con progranulina (PGRN). El rAAV comprende (a) una cápside del virus adenoasociado 1, y (b) un genoma del vector empaquetado en la cápside de AAV, en donde dicho genoma del vector comprende repeticiones terminales invertidas de AAV, una secuencia codificante para progranulina humana y secuencias reguladoras que dirigen la expresión de la progranulina; también se proporciona un método para tratar a un paciente humano con PGRN-FTD y otra neurodegeneración de inicio en adultos causada por haploinsuficiencias de granulina (GRN), que comprende administrar al sistema nervioso central (SNC) un virus adenoasociado recombinante (rAAV) que tiene una cápside del virus adenoasociado 1 (AAV1), en donde dicho rAAV comprende además un genoma del vector empaquetado en la cápside de AAV, en donde dicho genoma del vector comprende repeticiones terminales invertidas de AAV, una secuencia codificante para progranulina humana y secuencias reguladoras que dirigen la expresión de la progranulina.
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 |
---|---|
MX2021010134A true MX2021010134A (es) | 2021-09-23 |
Family
ID=70058449
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2021010134A MX2021010134A (es) | 2019-02-22 | 2020-02-21 | Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn. |
Country Status (16)
Country | Link |
---|---|
US (1) | US20220136008A1 (es) |
EP (1) | EP3927381A1 (es) |
JP (1) | JP2022523766A (es) |
KR (1) | KR20210131370A (es) |
CN (1) | CN113710281A (es) |
AU (1) | AU2020225472A1 (es) |
BR (1) | BR112021015817A2 (es) |
CA (1) | CA3129672A1 (es) |
CL (1) | CL2021002172A1 (es) |
CO (1) | CO2021011040A2 (es) |
IL (1) | IL285654A (es) |
MX (1) | MX2021010134A (es) |
PE (1) | PE20211819A1 (es) |
SG (1) | SG11202108504YA (es) |
TW (1) | TW202045730A (es) |
WO (1) | WO2020172490A1 (es) |
Families Citing this family (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2020160458A1 (en) * | 2019-02-01 | 2020-08-06 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
GB201913974D0 (en) * | 2019-09-27 | 2019-11-13 | King S College London | Vector |
WO2021081201A1 (en) * | 2019-10-22 | 2021-04-29 | Applied Genetic Technologies Corporation | Adeno-associated virus (aav) systems for treatment of progranulin associated neurodegeneative diseases or disorders |
IL300622A (en) * | 2020-08-26 | 2023-04-01 | Univ Pennsylvania | Recombinant adeno-associated virus for the treatment of adult-onset GRN-related neurodegeneration |
WO2023150506A2 (en) * | 2022-02-01 | 2023-08-10 | Shape Therapeutics Inc. | STABLE CELL LINES FOR INDUCIBLE PRODUCTION OF rAAV VIRIONS |
Family Cites Families (28)
Publication number | Priority date | Publication date | Assignee | Title |
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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 |
US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
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 |
ATE270324T1 (de) | 1997-04-14 | 2004-07-15 | Cell Genesys Inc | Methoden zur erhöhung der effizienz rekombinanter aav produkte |
EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol 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 |
DE69941905D1 (de) | 1998-11-10 | 2010-02-25 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
NZ522841A (en) | 2000-06-01 | 2004-12-24 | Univ North Carolina | Surgically implantable matrix with a viral vector dried onto it for controlled release of recombinant parvovirus vectors |
IL161827A0 (en) | 2001-11-13 | 2005-11-20 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby |
US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
PL2292780T3 (pl) | 2003-09-30 | 2018-05-30 | The Trustees Of The University Of Pennsylvania | Klady wirusów związanych z adenowirusami (AAV), sekwencje, wektory je zawierające i ich zastosowania |
DE23193606T1 (de) | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
CA3139726A1 (en) * | 2006-06-07 | 2007-12-21 | Genzyme Corporation | Gene therapy for motor disorders |
WO2009089635A1 (en) * | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
CN102869779A (zh) | 2010-03-29 | 2013-01-09 | 宾夕法尼亚大学托管会 | 药理学诱导的转基因消融*** |
FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
CN116019934A (zh) * | 2015-02-10 | 2023-04-28 | 建新公司 | 病毒颗粒至纹状体和皮质的增强递送 |
EP3387118B1 (en) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
US11015174B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV8 |
CA3016314A1 (en) * | 2016-03-02 | 2017-09-08 | Julianne REIDERS | Therapy for frontotemporal dementia |
CA3053399A1 (en) | 2017-02-28 | 2018-09-07 | The Trustees Of The University Of Pennsylvania | Novel adeno-associated virus (aav) clade f vector and uses therefor |
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2020
- 2020-02-21 JP JP2021549291A patent/JP2022523766A/ja active Pending
- 2020-02-21 BR BR112021015817-0A patent/BR112021015817A2/pt unknown
- 2020-02-21 PE PE2021001357A patent/PE20211819A1/es unknown
- 2020-02-21 KR KR1020217029575A patent/KR20210131370A/ko unknown
- 2020-02-21 SG SG11202108504YA patent/SG11202108504YA/en unknown
- 2020-02-21 AU AU2020225472A patent/AU2020225472A1/en active Pending
- 2020-02-21 EP EP20715985.6A patent/EP3927381A1/en active Pending
- 2020-02-21 TW TW109105680A patent/TW202045730A/zh unknown
- 2020-02-21 CN CN202080015962.8A patent/CN113710281A/zh active Pending
- 2020-02-21 WO PCT/US2020/019149 patent/WO2020172490A1/en unknown
- 2020-02-21 MX MX2021010134A patent/MX2021010134A/es unknown
- 2020-02-21 US US17/431,937 patent/US20220136008A1/en active Pending
- 2020-02-21 CA CA3129672A patent/CA3129672A1/en active Pending
-
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
Also Published As
Publication number | Publication date |
---|---|
CN113710281A (zh) | 2021-11-26 |
CL2021002172A1 (es) | 2022-02-11 |
EP3927381A1 (en) | 2021-12-29 |
CA3129672A1 (en) | 2020-08-27 |
WO2020172490A1 (en) | 2020-08-27 |
JP2022523766A (ja) | 2022-04-26 |
CO2021011040A2 (es) | 2021-09-09 |
PE20211819A1 (es) | 2021-09-14 |
KR20210131370A (ko) | 2021-11-02 |
TW202045730A (zh) | 2020-12-16 |
SG11202108504YA (en) | 2021-09-29 |
AU2020225472A1 (en) | 2021-08-26 |
BR112021015817A2 (pt) | 2021-10-13 |
IL285654A (en) | 2021-09-30 |
US20220136008A1 (en) | 2022-05-05 |
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