PE20211819A1 - 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 grnInfo
- Publication number
- PE20211819A1 PE20211819A1 PE2021001357A PE2021001357A PE20211819A1 PE 20211819 A1 PE20211819 A1 PE 20211819A1 PE 2021001357 A PE2021001357 A PE 2021001357A PE 2021001357 A PE2021001357 A PE 2021001357A PE 20211819 A1 PE20211819 A1 PE 20211819A1
- Authority
- PE
- Peru
- Prior art keywords
- progranulin
- aav
- grn
- associated virus
- capsid
- 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
-
- 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)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- General Chemical & Material Sciences (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hospice & Palliative Care (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Psychology (AREA)
- Orthopedic Medicine & Surgery (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 neurodegeneracion 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 capside del virus adenoasociado 1, y (b) un genoma del vector empaquetado en la capside 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 expresion de la progranulina. Tambien se proporciona un metodo para tratar a un paciente humano con PGRN-FTD y otra neurodegeneracion 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 capside del virus adenoasociado 1 (AAV1), en donde dicho rAAV comprende ademas un genoma del vector empaquetado en la capside 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 expresion 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 |
---|---|
PE20211819A1 true PE20211819A1 (es) | 2021-09-14 |
Family
ID=70058449
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PE2021001357A PE20211819A1 (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 |
---|---|---|---|---|
CA3128003A1 (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 |
EP4048799A4 (en) * | 2019-10-22 | 2023-11-15 | Applied Genetic Technologies Corporation | ADENO-ASSOCIATED VIRUS (AAV) SYSTEMS FOR THE TREATMENT OF PROGRANULIN-ASSOCIATED NEURODEGENERATIVE DISEASES OR DISORDERS |
TW202227633A (zh) * | 2020-08-26 | 2022-07-16 | 賓州大學委員會 | 用於治療grn相關的成年發病神經退化之重組腺相關病毒 |
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 |
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 |
CA2287478C (en) | 1997-04-14 | 2007-06-19 | Richard J. Samulski | 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 |
DE69941905D1 (de) | 1998-11-10 | 2010-02-25 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
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 |
CN103555677B (zh) | 2001-11-13 | 2018-01-30 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
JP5054975B2 (ja) | 2003-09-30 | 2012-10-24 | 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 | アデノ随伴ウイルス(aav)の同源系統群、配列、それらを含有するベクターおよびそれらの用途 |
EP3603676A1 (en) | 2005-04-07 | 2020-02-05 | The Trustees of the University of Pennsylvania | Method of increasing the function of an aav vector |
EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
BRPI0711965A2 (pt) * | 2006-06-07 | 2012-01-24 | Genzyme Corp | terapia de gene para esclerose lateral amiotrófica e outras enfermidades da medula espinal |
CN102006882B (zh) * | 2008-01-16 | 2017-06-06 | 神经动力公司 | 利用颗粒体蛋白前体(pgrn)治疗神经变性疾病 |
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 |
MX2017010370A (es) * | 2015-02-10 | 2017-12-14 | Genzyme Corp | Suministro mejorado de particulas virales al cuerpo estriado y al cortex. |
US11015174B2 (en) | 2015-12-11 | 2021-05-25 | 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 |
WO2017160360A2 (en) | 2015-12-11 | 2017-09-21 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
WO2017100704A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
WO2017151884A1 (en) * | 2016-03-02 | 2017-09-08 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
KR20190132639A (ko) | 2017-02-28 | 2019-11-28 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 아데노-연관 바이러스(aav) 클레이드 f 벡터 및 이의 용도 |
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2020
- 2020-02-21 EP EP20715985.6A patent/EP3927381A1/en active Pending
- 2020-02-21 KR KR1020217029575A patent/KR20210131370A/ko unknown
- 2020-02-21 MX MX2021010134A patent/MX2021010134A/es unknown
- 2020-02-21 TW TW109105680A patent/TW202045730A/zh unknown
- 2020-02-21 WO PCT/US2020/019149 patent/WO2020172490A1/en unknown
- 2020-02-21 CA CA3129672A patent/CA3129672A1/en active Pending
- 2020-02-21 PE PE2021001357A patent/PE20211819A1/es unknown
- 2020-02-21 CN CN202080015962.8A patent/CN113710281A/zh active Pending
- 2020-02-21 US US17/431,937 patent/US20220136008A1/en active Pending
- 2020-02-21 BR BR112021015817-0A patent/BR112021015817A2/pt unknown
- 2020-02-21 SG SG11202108504YA patent/SG11202108504YA/en unknown
- 2020-02-21 AU AU2020225472A patent/AU2020225472A1/en active Pending
- 2020-02-21 JP JP2021549291A patent/JP2022523766A/ja active Pending
-
2021
- 2021-08-16 CL CL2021002172A patent/CL2021002172A1/es unknown
- 2021-08-16 IL IL285654A patent/IL285654A/en unknown
- 2021-08-23 CO CONC2021/0011040A patent/CO2021011040A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
US20220136008A1 (en) | 2022-05-05 |
KR20210131370A (ko) | 2021-11-02 |
CA3129672A1 (en) | 2020-08-27 |
CN113710281A (zh) | 2021-11-26 |
TW202045730A (zh) | 2020-12-16 |
BR112021015817A2 (pt) | 2021-10-13 |
AU2020225472A1 (en) | 2021-08-26 |
SG11202108504YA (en) | 2021-09-29 |
MX2021010134A (es) | 2021-09-23 |
JP2022523766A (ja) | 2022-04-26 |
EP3927381A1 (en) | 2021-12-29 |
CO2021011040A2 (es) | 2021-09-09 |
CL2021002172A1 (es) | 2022-02-11 |
WO2020172490A1 (en) | 2020-08-27 |
IL285654A (en) | 2021-09-30 |
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