SG11202108504YA - Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration - Google Patents
Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegenerationInfo
- Publication number
- SG11202108504YA SG11202108504YA SG11202108504YA SG11202108504YA SG11202108504YA SG 11202108504Y A SG11202108504Y A SG 11202108504YA SG 11202108504Y A SG11202108504Y A SG 11202108504YA SG 11202108504Y A SG11202108504Y A SG 11202108504YA SG 11202108504Y A SG11202108504Y A SG 11202108504YA
- Authority
- SG
- Singapore
- Prior art keywords
- grn
- treatment
- recombinant adeno
- adult
- associated virus
- 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
-
- 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)
- 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)
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 |
|---|---|
| SG11202108504YA true SG11202108504YA (en) | 2021-09-29 |
Family
ID=70058449
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11202108504YA SG11202108504YA (en) | 2019-02-22 | 2020-02-21 | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20220136008A1 (en) |
| EP (1) | EP3927381A1 (en) |
| JP (1) | JP2022523766A (en) |
| KR (1) | KR20210131370A (en) |
| CN (1) | CN113710281A (en) |
| AU (1) | AU2020225472A1 (en) |
| BR (1) | BR112021015817A2 (en) |
| CA (1) | CA3129672A1 (en) |
| CL (1) | CL2021002172A1 (en) |
| CO (1) | CO2021011040A2 (en) |
| IL (1) | IL285654A (en) |
| MX (1) | MX2021010134A (en) |
| PE (1) | PE20211819A1 (en) |
| SG (1) | SG11202108504YA (en) |
| TW (1) | TW202045730A (en) |
| WO (1) | WO2020172490A1 (en) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3917539A4 (en) * | 2019-02-01 | 2022-11-23 | 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 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 |
| 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 |
|---|---|---|---|---|
| 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 |
| DE69824859T2 (en) | 1997-04-14 | 2005-08-04 | Cell Genesys, Inc., Foster City | METHODS OF INCREASING THE EFFICIENCY OF RECOMBINANT AAV PRODUCTS |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| 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 (en) | 1998-11-10 | 2010-02-25 | Univ North Carolina | VIRUS VECTORS AND METHOD FOR THEIR MANUFACTURE AND ADMINISTRATION. |
| 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 |
| BRPI0214119B8 (en) | 2001-11-13 | 2021-05-25 | Univ Pennsylvania | recombinant adeno-associated virus, method of generating said virus and composition comprising said virus |
| US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
| PT3211085T (en) | 2003-09-30 | 2021-06-17 | Univ Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| CN101203613B (en) | 2005-04-07 | 2012-12-12 | 宾夕法尼亚大学托管会 | Method of increasing the function of gland related viral vector |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| ES2725552T3 (en) * | 2006-06-07 | 2019-09-24 | Genzyme Corp | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
| US20100324127A1 (en) * | 2008-01-16 | 2010-12-23 | Kay Denis G | Treating neurodegenerative diseases with progranulin |
| CN102869779A (en) | 2010-03-29 | 2013-01-09 | 宾夕法尼亚大学托管会 | Pharmacologically induced transgene ablation system |
| FR2977562B1 (en) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | SEALED AND THERMALLY INSULATING TANK INTEGRATED IN A CARRIER STRUCTURE |
| PT3256594T (en) * | 2015-02-10 | 2022-01-18 | Genzyme Corp | Enhanced delivery of viral particles to the striatum and cortex |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| ES2918998T3 (en) | 2015-12-11 | 2022-07-21 | Univ 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 |
| US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
| JP7436089B2 (en) * | 2016-03-02 | 2024-02-21 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア | Treatment of 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 |
-
2020
- 2020-02-21 JP JP2021549291A patent/JP2022523766A/en active Pending
- 2020-02-21 MX MX2021010134A patent/MX2021010134A/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/en unknown
- 2020-02-21 KR KR1020217029575A patent/KR20210131370A/en 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 BR BR112021015817-0A patent/BR112021015817A2/en unknown
- 2020-02-21 SG SG11202108504YA patent/SG11202108504YA/en unknown
- 2020-02-21 PE PE2021001357A patent/PE20211819A1/en unknown
- 2020-02-21 US US17/431,937 patent/US20220136008A1/en active Pending
- 2020-02-21 CN CN202080015962.8A patent/CN113710281A/en active Pending
-
2021
- 2021-08-16 CL CL2021002172A patent/CL2021002172A1/en unknown
- 2021-08-16 IL IL285654A patent/IL285654A/en unknown
- 2021-08-23 CO CONC2021/0011040A patent/CO2021011040A2/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CA3129672A1 (en) | 2020-08-27 |
| US20220136008A1 (en) | 2022-05-05 |
| MX2021010134A (en) | 2021-09-23 |
| EP3927381A1 (en) | 2021-12-29 |
| TW202045730A (en) | 2020-12-16 |
| CL2021002172A1 (en) | 2022-02-11 |
| BR112021015817A2 (en) | 2021-10-13 |
| IL285654A (en) | 2021-09-30 |
| CO2021011040A2 (en) | 2021-09-09 |
| WO2020172490A1 (en) | 2020-08-27 |
| PE20211819A1 (en) | 2021-09-14 |
| KR20210131370A (en) | 2021-11-02 |
| AU2020225472A1 (en) | 2021-08-26 |
| JP2022523766A (en) | 2022-04-26 |
| CN113710281A (en) | 2021-11-26 |
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