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
-
- 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)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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 |
|---|---|
| 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) | 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 |
|---|---|---|---|---|
| US20220111005A1 (en) * | 2019-02-01 | 2022-04-14 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| CA3129672A1 (en) | 2019-02-22 | 2020-08-27 | The Trustees Of The University Of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| 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 |
| MX2023002364A (es) * | 2020-08-26 | 2023-05-22 | Univ Pennsylvania | Virus adenoasociado recombinante para el tratamiento de la neurodegeneración iniciada en adultos asociada a grn. |
| JPWO2022131322A1 (enExample) * | 2020-12-17 | 2022-06-23 | ||
| EP4473123A2 (en) * | 2022-02-01 | 2024-12-11 | 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)
| 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 |
| AU728220B2 (en) | 1997-04-14 | 2001-01-04 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
| EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
| EP1127150B1 (en) | 1998-11-05 | 2007-05-16 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| 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 |
| WO2000028004A1 (en) | 1998-11-10 | 2000-05-18 | The University Of North Carolina At Chapel Hill | 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 |
| MX346493B (es) | 2001-11-13 | 2017-03-21 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| US20070015238A1 (en) | 2002-06-05 | 2007-01-18 | Snyder Richard O | Production of pseudotyped recombinant AAV virions |
| CN102199626B (zh) | 2003-09-30 | 2015-06-24 | 宾夕法尼亚大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| EP2383346B1 (en) | 2005-04-07 | 2014-10-08 | The Trustees of the University of Pennsylvania | Modified AAVrh.64 capsids, compositions containing same and uses thereof |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| WO2008019187A2 (en) | 2006-05-30 | 2008-02-14 | Mayo Foundation For Medical Education And Research | Detecting and treating dementia |
| WO2007146046A2 (en) * | 2006-06-07 | 2007-12-21 | Genzyme Corporation | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
| 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 |
| LT4342992T (lt) | 2009-05-02 | 2025-08-25 | Genzyme Corporation | Genų terapija, skirta neurodegeneracinių sutrikimų gydymui |
| 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 |
| EP2553106A2 (en) | 2010-03-29 | 2013-02-06 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| CA2818253A1 (en) | 2010-11-16 | 2012-05-24 | 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 |
| KR102863734B1 (ko) * | 2015-02-10 | 2025-09-25 | 젠자임 코포레이션 | 선조체 및 피질로의 바이러스 입자의 향상된 전달 |
| MX2018004755A (es) * | 2015-10-29 | 2018-12-19 | Voyager Therapeutics Inc | Entrega de polinucleotidos dirigidos al sistema nervioso central. |
| US20190060359A1 (en) | 2015-11-02 | 2019-02-28 | Neuraltus Pharmaceuticals, Inc. | Treatment of neurodegenerative disease with sodium chlorite |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | 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 |
| EP3387118B1 (en) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aavrh10 |
| MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de 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 |
| MX2019002518A (es) | 2016-09-02 | 2019-07-18 | Spark Therapeutics Inc | Metodos y vectores para el tratamiento de trastornos del snc. |
| CN118956781A (zh) | 2017-02-28 | 2024-11-15 | 宾夕法尼亚州大学信托人 | 腺相关病毒(aav)进化枝f载体及其用途 |
| KR20220010062A (ko) | 2017-10-03 | 2022-01-25 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| KR102709597B1 (ko) | 2017-10-03 | 2024-09-26 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애를 위한 유전자 요법 |
| US20200283800A1 (en) | 2017-10-23 | 2020-09-10 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative diseases |
| US20220111005A1 (en) | 2019-02-01 | 2022-04-14 | Avrobio, Inc. | Compositions and methods for treating neurocognitive disorders |
| CA3129672A1 (en) | 2019-02-22 | 2020-08-27 | The Trustees Of The University Of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| MX2023002364A (es) | 2020-08-26 | 2023-05-22 | Univ Pennsylvania | Virus adenoasociado recombinante para el tratamiento de la neurodegeneración iniciada en adultos asociada a grn. |
-
2020
- 2020-02-21 CA CA3129672A patent/CA3129672A1/en active Pending
- 2020-02-21 TW TW109105680A patent/TW202045730A/zh unknown
- 2020-02-21 AU AU2020225472A patent/AU2020225472A1/en active Pending
- 2020-02-21 BR BR112021015817-0A patent/BR112021015817A2/pt unknown
- 2020-02-21 WO PCT/US2020/019149 patent/WO2020172490A1/en not_active Ceased
- 2020-02-21 PE PE2021001357A patent/PE20211819A1/es unknown
- 2020-02-21 JP JP2021549291A patent/JP7637058B2/ja active Active
- 2020-02-21 KR KR1020217029575A patent/KR20210131370A/ko active Pending
- 2020-02-21 CN CN202080015962.8A patent/CN113710281A/zh active Pending
- 2020-02-21 US US17/431,937 patent/US12416017B2/en active Active
- 2020-02-21 MX MX2021010134A patent/MX2021010134A/es unknown
- 2020-02-21 EP EP20715985.6A patent/EP3927381A1/en active Pending
- 2020-02-21 SG SG11202108504YA patent/SG11202108504YA/en unknown
-
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 |
|---|---|
| CN113710281A (zh) | 2021-11-26 |
| CL2021002172A1 (es) | 2022-02-11 |
| MX2021010134A (es) | 2021-09-23 |
| KR20210131370A (ko) | 2021-11-02 |
| EP3927381A1 (en) | 2021-12-29 |
| AU2020225472A1 (en) | 2021-08-26 |
| CA3129672A1 (en) | 2020-08-27 |
| CO2021011040A2 (es) | 2021-09-09 |
| US12416017B2 (en) | 2025-09-16 |
| BR112021015817A2 (pt) | 2021-10-13 |
| JP2025081439A (ja) | 2025-05-27 |
| JP7637058B2 (ja) | 2025-02-27 |
| JP2022523766A (ja) | 2022-04-26 |
| SG11202108504YA (en) | 2021-09-29 |
| US20220136008A1 (en) | 2022-05-05 |
| WO2020172490A1 (en) | 2020-08-27 |
| IL285654A (en) | 2021-09-30 |
| TW202045730A (zh) | 2020-12-16 |
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