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
- 241000702421 Dependoparvovirus Species 0.000 title 1
- 230000004770 neurodegeneration Effects 0.000 title 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)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (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)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hospice & Palliative Care (AREA)
- General Chemical & Material Sciences (AREA)
- Psychiatry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Immunology (AREA)
- Psychology (AREA)
- Orthopedic Medicine & Surgery (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) | US12416017B2 (enExample) |
| EP (1) | EP3927381A1 (enExample) |
| JP (2) | JP7637058B2 (enExample) |
| KR (1) | KR20210131370A (enExample) |
| CN (1) | CN113710281A (enExample) |
| AU (1) | AU2020225472A1 (enExample) |
| BR (1) | BR112021015817A2 (enExample) |
| CA (1) | CA3129672A1 (enExample) |
| CL (1) | CL2021002172A1 (enExample) |
| CO (1) | CO2021011040A2 (enExample) |
| IL (1) | IL285654A (enExample) |
| MX (1) | MX2021010134A (enExample) |
| PE (1) | PE20211819A1 (enExample) |
| SG (1) | SG11202108504YA (enExample) |
| TW (1) | TW202045730A (enExample) |
| WO (1) | WO2020172490A1 (enExample) |
Families Citing this family (10)
| 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 |
| JP7637058B2 (ja) | 2019-02-22 | 2025-02-27 | 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 | Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
| GB201913974D0 (en) * | 2019-09-27 | 2019-11-13 | King S College London | Vector |
| MX2022004812A (es) * | 2019-10-22 | 2023-02-23 | Applied Genetic Tech Corporation | Sistemas de virus adeno-asociados (aav) para el tratamiento de enfermedades o trastornos neurodegenerativos asociados a la progranulina. |
| CN116887867A (zh) * | 2020-08-26 | 2023-10-13 | 宾夕法尼亚州大学信托人 | 用于治疗grn相关成人发作性神经退行性病变的重组腺相关病毒 |
| EP4265270A4 (en) * | 2020-12-17 | 2025-02-26 | Kyoto University | NEURON ACTIVATOR |
| WO2023150506A2 (en) * | 2022-02-01 | 2023-08-10 | Shape Therapeutics Inc. | STABLE CELL LINES FOR INDUCIBLE PRODUCTION OF rAAV VIRIONS |
| WO2024245120A1 (en) * | 2023-05-26 | 2024-12-05 | Shanghai Vitalgen Biopharma Co., Ltd. | Recombinant aav vectors for treating proteinopathies in central nervous system |
| AU2024311750A1 (en) * | 2023-06-21 | 2025-12-18 | Eli Lilly And Company | Compounds for the delivery of granulin across the blood brain barrier |
| WO2025188625A1 (en) * | 2024-03-03 | 2025-09-12 | Passage Bio, Inc. | Recombinant adeno-associated virus for treatment of neurodegenerative disorders |
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 |
| 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 |
| NZ500546A (en) | 1997-04-14 | 2001-09-28 | Cell Genesys Inc | Methods for increasing the production of high titre stocks of recombinant adeno-associated virus (AAV) |
| 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 |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| JP2002538770A (ja) | 1998-11-10 | 2002-11-19 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | ウイルスベクターとその製造及び投与の方法 |
| AU2001268149B2 (en) | 2000-06-01 | 2005-08-18 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| ES2258601T3 (es) | 2001-11-13 | 2006-09-01 | The Trustees Of The University Of Pennsylvania | Un metodo para la identificacion de las secuencias desconocidas del virus adeno-asociado (vaa) y un kit para el metodo. |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP3910063A1 (en) | 2003-09-30 | 2021-11-17 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| EP4234687A2 (en) | 2005-04-07 | 2023-08-30 | 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 |
| CA2653974A1 (en) | 2006-05-30 | 2008-02-14 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
| PL2489733T3 (pl) * | 2006-06-07 | 2019-08-30 | Genzyme Corporation | Terapia genowa w stwardnieniu zanikowym bocznym i innych zaburzeniach rdzenia kręgowego |
| EP3009143B1 (en) | 2008-01-16 | 2018-10-03 | Neurodyn Life Sciences Inc. | Progranulin for use in treating parkinson's disease or alzheimer's disease |
| BRPI1010868A2 (pt) | 2009-05-02 | 2018-06-12 | Genzyme Corp | terapia gênica para distúrbios neurodegenerativos |
| 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 |
| MX342858B (es) | 2010-03-29 | 2016-10-13 | The Trustees Of The Univ Of Pennsylvania * | Sistema de ablacion transgenica inducida farmacologicamente. |
| US20150352185A1 (en) | 2010-11-16 | 2015-12-10 | Denis G. Kay | Method for increasing neprilysin expression and activity |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| 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 |
| PL3256594T3 (pl) * | 2015-02-10 | 2022-02-14 | Genzyme Corporation | Ulepszone dostarczanie cząstek wirusa do prążkowia i kory |
| AU2016343979A1 (en) * | 2015-10-29 | 2018-05-10 | Voyager Therapeutics, Inc. | Delivery of central nervous system targeting polynucleotides |
| US20190060359A1 (en) | 2015-11-02 | 2019-02-28 | Neuraltus Pharmaceuticals, Inc. | Treatment of neurodegenerative disease with sodium chlorite |
| EP4215605A1 (en) | 2015-12-11 | 2023-07-26 | The Trustees of The University of Pennsylvania | Scalable purification method for aav8 |
| US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| CA3012195A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| CA3016314A1 (en) | 2016-03-02 | 2017-09-08 | Julianne REIDERS | 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 |
| FI3589730T3 (fi) | 2017-02-28 | 2024-02-22 | Univ Pennsylvania | Kladin f adenoassosioitunut virus (aav) -vektori ja sen käyttötapoja |
| KR102697811B1 (ko) | 2017-10-03 | 2024-08-22 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| CA3078501A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| US20200283800A1 (en) | 2017-10-23 | 2020-09-10 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| CA3128003A1 (en) | 2019-02-01 | 2020-08-06 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| JP7637058B2 (ja) | 2019-02-22 | 2025-02-27 | 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 | Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
| CN116887867A (zh) | 2020-08-26 | 2023-10-13 | 宾夕法尼亚州大学信托人 | 用于治疗grn相关成人发作性神经退行性病变的重组腺相关病毒 |
-
2020
- 2020-02-21 JP JP2021549291A patent/JP7637058B2/ja active Active
- 2020-02-21 US US17/431,937 patent/US12416017B2/en active Active
- 2020-02-21 TW TW109105680A patent/TW202045730A/zh unknown
- 2020-02-21 WO PCT/US2020/019149 patent/WO2020172490A1/en not_active Ceased
- 2020-02-21 AU AU2020225472A patent/AU2020225472A1/en active Pending
- 2020-02-21 CN CN202080015962.8A patent/CN113710281A/zh active Pending
- 2020-02-21 PE PE2021001357A patent/PE20211819A1/es unknown
- 2020-02-21 KR KR1020217029575A patent/KR20210131370A/ko active Pending
- 2020-02-21 EP EP20715985.6A patent/EP3927381A1/en active Pending
- 2020-02-21 SG SG11202108504YA patent/SG11202108504YA/en unknown
- 2020-02-21 BR BR112021015817-0A patent/BR112021015817A2/pt unknown
- 2020-02-21 MX MX2021010134A patent/MX2021010134A/es unknown
- 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
-
2025
- 2025-02-14 JP JP2025022078A patent/JP2025081439A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| TW202045730A (zh) | 2020-12-16 |
| KR20210131370A (ko) | 2021-11-02 |
| CN113710281A (zh) | 2021-11-26 |
| WO2020172490A1 (en) | 2020-08-27 |
| AU2020225472A1 (en) | 2021-08-26 |
| CA3129672A1 (en) | 2020-08-27 |
| JP2025081439A (ja) | 2025-05-27 |
| JP7637058B2 (ja) | 2025-02-27 |
| US12416017B2 (en) | 2025-09-16 |
| US20220136008A1 (en) | 2022-05-05 |
| CL2021002172A1 (es) | 2022-02-11 |
| EP3927381A1 (en) | 2021-12-29 |
| MX2021010134A (es) | 2021-09-23 |
| BR112021015817A2 (pt) | 2021-10-13 |
| CO2021011040A2 (es) | 2021-09-09 |
| PE20211819A1 (es) | 2021-09-14 |
| IL285654A (en) | 2021-09-30 |
| JP2022523766A (ja) | 2022-04-26 |
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