BR112023002904A2 - Vírus adenoassociado recombinante para tratar neurodegeneração com início na idade adulta associada a grn - Google Patents
Vírus adenoassociado recombinante para tratar neurodegeneração com início na idade adulta associada a grnInfo
- Publication number
- BR112023002904A2 BR112023002904A2 BR112023002904A BR112023002904A BR112023002904A2 BR 112023002904 A2 BR112023002904 A2 BR 112023002904A2 BR 112023002904 A BR112023002904 A BR 112023002904A BR 112023002904 A BR112023002904 A BR 112023002904A BR 112023002904 A2 BR112023002904 A2 BR 112023002904A2
- Authority
- BR
- Brazil
- Prior art keywords
- grn
- onceive
- recombinant adeno
- associated virus
- treat adult
- Prior art date
Links
- 230000004770 neurodegeneration Effects 0.000 title abstract 4
- 241000702421 Dependoparvovirus Species 0.000 title abstract 3
- 239000013607 AAV vector Substances 0.000 abstract 2
- 102100037632 Progranulin Human genes 0.000 abstract 2
- 208000015122 neurodegenerative disease Diseases 0.000 abstract 2
- 239000013598 vector Substances 0.000 abstract 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 abstract 1
- 102000019204 Progranulins Human genes 0.000 abstract 1
- 108010012809 Progranulins Proteins 0.000 abstract 1
- 210000000234 capsid Anatomy 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 238000011285 therapeutic regimen Methods 0.000 abstract 1
Classifications
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0016—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/18—Growth factors; Growth regulators
-
- 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
- A61K48/0083—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 administration regime
-
- 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
- C07K14/475—Growth factors; Growth regulators
-
- 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
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
- A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
-
- 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
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Neurosurgery (AREA)
- Wood Science & Technology (AREA)
- Neurology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hospice & Palliative Care (AREA)
- Microbiology (AREA)
- Psychiatry (AREA)
- Virology (AREA)
- Toxicology (AREA)
- Immunology (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 |
|---|---|---|---|
| US202063070639P | 2020-08-26 | 2020-08-26 | |
| PCT/US2021/047686 WO2022046988A1 (en) | 2020-08-26 | 2021-08-26 | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112023002904A2 true BR112023002904A2 (pt) | 2023-04-25 |
Family
ID=78032503
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112023002904A BR112023002904A2 (pt) | 2020-08-26 | 2021-08-26 | Vírus adenoassociado recombinante para tratar neurodegeneração com início na idade adulta associada a grn |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20230364264A1 (https=) |
| EP (1) | EP4204014A1 (https=) |
| JP (1) | JP2023540054A (https=) |
| KR (1) | KR20230058102A (https=) |
| CN (1) | CN116887867A (https=) |
| AR (1) | AR123358A1 (https=) |
| AU (1) | AU2021332331A1 (https=) |
| BR (1) | BR112023002904A2 (https=) |
| CA (1) | CA3189107A1 (https=) |
| IL (1) | IL300622A (https=) |
| MX (1) | MX2023002364A (https=) |
| TW (1) | TW202227633A (https=) |
| WO (1) | WO2022046988A1 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US20250295807A1 (en) * | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2024035649A1 (en) * | 2022-08-08 | 2024-02-15 | Shape Therapeutics Inc. | Compositions for progranulin expression and methods of use thereof |
| GB202216168D0 (en) | 2022-10-31 | 2022-12-14 | UCB Biopharma SRL | Route of administration |
| WO2024191873A1 (en) * | 2023-03-10 | 2024-09-19 | Biogen Ma Inc. | Packaging plasmids for aav production |
| 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 |
| WO2025231406A1 (en) * | 2024-05-02 | 2025-11-06 | The Children's Hospital Of Philadelphia | Methods to increase transduction of ependyma cells in brain |
Family Cites Families (27)
| 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 |
| 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 |
| US6548286B1 (en) | 1997-04-14 | 2003-04-15 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
| 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 |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| 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 |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
| ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | 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 |
| CA3012195A1 (en) * | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| US20190328906A1 (en) * | 2016-03-02 | 2019-10-31 | The Children's Hospital Of Philadelphia | Therapy for frontotemporal dementia |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| 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 |
-
2021
- 2021-08-26 EP EP21783625.3A patent/EP4204014A1/en active Pending
- 2021-08-26 JP JP2023513708A patent/JP2023540054A/ja active Pending
- 2021-08-26 WO PCT/US2021/047686 patent/WO2022046988A1/en not_active Ceased
- 2021-08-26 TW TW110131628A patent/TW202227633A/zh unknown
- 2021-08-26 CN CN202180072913.2A patent/CN116887867A/zh active Pending
- 2021-08-26 KR KR1020237010072A patent/KR20230058102A/ko active Pending
- 2021-08-26 US US18/040,971 patent/US20230364264A1/en active Pending
- 2021-08-26 CA CA3189107A patent/CA3189107A1/en active Pending
- 2021-08-26 AR ARP210102410A patent/AR123358A1/es unknown
- 2021-08-26 BR BR112023002904A patent/BR112023002904A2/pt unknown
- 2021-08-26 AU AU2021332331A patent/AU2021332331A1/en active Pending
- 2021-08-26 IL IL300622A patent/IL300622A/en unknown
- 2021-08-26 MX MX2023002364A patent/MX2023002364A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230058102A (ko) | 2023-05-02 |
| EP4204014A1 (en) | 2023-07-05 |
| AU2021332331A1 (en) | 2023-03-09 |
| MX2023002364A (es) | 2023-05-22 |
| TW202227633A (zh) | 2022-07-16 |
| CA3189107A1 (en) | 2022-03-03 |
| CN116887867A (zh) | 2023-10-13 |
| AR123358A1 (es) | 2022-11-23 |
| WO2022046988A1 (en) | 2022-03-03 |
| IL300622A (en) | 2023-04-01 |
| JP2023540054A (ja) | 2023-09-21 |
| US20230364264A1 (en) | 2023-11-16 |
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