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
-
- 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)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Immunology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Psychology (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 |
---|---|---|---|---|
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 |
KR20230019402A (en) * | 2019-10-22 | 2023-02-08 | 어플라이드 제네틱스 테크놀로지스 코퍼레이션 | Adeno-associated virus (AAV) system for the treatment of progranulin associated neurodegenerative diseases or disorders |
US20230364264A1 (en) * | 2020-08-26 | 2023-11-16 | 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 |
ES2224375T3 (en) | 1997-04-14 | 2005-03-01 | Cell Genesys, Inc. | METHODS TO INCREASE THE EFFECTIVENESS OF THE 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 |
EP1135468B1 (en) | 1998-11-10 | 2010-01-06 | University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
DE60139471D1 (en) | 2000-06-01 | 2009-09-17 | Univ North Carolina | METHOD AND COMPOSITIONS FOR CONTROLLED DISPOSAL OF RECOMBINANT PARVOVIRUS VECTORS |
NZ600121A (en) | 2001-11-13 | 2013-12-20 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
ES2874298T3 (en) | 2003-09-30 | 2021-11-04 | Univ Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing the same, and uses thereof |
US8999678B2 (en) | 2005-04-07 | 2015-04-07 | 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 |
MX364444B (en) * | 2006-06-07 | 2019-04-26 | Genzyme Corp | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders. |
WO2009089635A1 (en) * | 2008-01-16 | 2009-07-23 | Neurodyn, Inc. | Treating neurodegenerative diseases with progranulin (pgrn) |
SG183929A1 (en) | 2010-03-29 | 2012-10-30 | Univ Pennsylvania | 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 |
SI3256594T1 (en) * | 2015-02-10 | 2022-07-29 | Genzyme Corporation | Enhanced delivery of viral particles to the striatum and cortex |
US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
EP4215605A1 (en) | 2015-12-11 | 2023-07-26 | The Trustees of The University of Pennsylvania | Scalable purification method for aav8 |
EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
ES2918998T3 (en) | 2015-12-11 | 2022-07-21 | Univ Pennsylvania | Scalable purification method for AAVrh10 |
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 SG SG11202108504YA patent/SG11202108504YA/en unknown
- 2020-02-21 CA CA3129672A patent/CA3129672A1/en active Pending
- 2020-02-21 CN CN202080015962.8A patent/CN113710281A/en active Pending
- 2020-02-21 KR KR1020217029575A patent/KR20210131370A/en unknown
- 2020-02-21 AU AU2020225472A patent/AU2020225472A1/en active Pending
- 2020-02-21 PE PE2021001357A patent/PE20211819A1/en unknown
- 2020-02-21 EP EP20715985.6A patent/EP3927381A1/en active Pending
- 2020-02-21 JP JP2021549291A patent/JP2022523766A/en active Pending
- 2020-02-21 TW TW109105680A patent/TW202045730A/en unknown
- 2020-02-21 BR BR112021015817-0A patent/BR112021015817A2/en unknown
- 2020-02-21 MX MX2021010134A patent/MX2021010134A/en unknown
- 2020-02-21 US US17/431,937 patent/US20220136008A1/en active Pending
- 2020-02-21 WO PCT/US2020/019149 patent/WO2020172490A1/en active Application Filing
-
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 |
---|---|
JP2022523766A (en) | 2022-04-26 |
BR112021015817A2 (en) | 2021-10-13 |
AU2020225472A1 (en) | 2021-08-26 |
MX2021010134A (en) | 2021-09-23 |
EP3927381A1 (en) | 2021-12-29 |
KR20210131370A (en) | 2021-11-02 |
CL2021002172A1 (en) | 2022-02-11 |
IL285654A (en) | 2021-09-30 |
CA3129672A1 (en) | 2020-08-27 |
TW202045730A (en) | 2020-12-16 |
CO2021011040A2 (en) | 2021-09-09 |
PE20211819A1 (en) | 2021-09-14 |
US20220136008A1 (en) | 2022-05-05 |
CN113710281A (en) | 2021-11-26 |
WO2020172490A1 (en) | 2020-08-27 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
IL285654A (en) | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration | |
SG11202103102WA (en) | Adeno-associated virus compositions for targeted gene therapy | |
IL262353A (en) | Methods of enhancing biological potency of baculovirus system-produced recombinant adeno-associated virus | |
EP3880823A4 (en) | Therapeutic adeno-associated virus for treating pompe disease | |
EP3990031A4 (en) | Adeno-associated virus purification methods | |
ZA202100859B (en) | Recombinant protein variants | |
EP3500278A4 (en) | Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus | |
IL288937A (en) | Improved treatment using eyp001 | |
IL290287A (en) | Exon 44-targeted nucleic acids and recombinant adeno-associated virus comprising said nucleic acids for treatment of dystrophin-based myopathies | |
IL285328A (en) | Adeno-associated virus delivery of cln3 polynucleotide | |
IL285329A (en) | Adeno-associated virus delivery of cln6 polynucleotide | |
EP4061167C0 (en) | Proctective hood for medical use | |
EP3503928A4 (en) | Methods and compositions for treating equine conditions using recombinant self-complementary adeno-associated virus | |
EP3440225A4 (en) | Therapeutic antibodies for treatment of neurodegeneration | |
IL282519A (en) | Peptide fragments for treatment of diabetes | |
SG11201704919SA (en) | Methods of purifying adeno-associated virus (aav) and/or recombinant adeno-associated virus (raav) | |
GB201803197D0 (en) | Viral treatment | |
EP4054606A4 (en) | Use of mbv for treating autoimmune diseae | |
IL279078A (en) | Combination therapy for treating hepatitis b virus infection | |
GB201916428D0 (en) | Treatment of hydro-carbon-contaminated materials | |
EP3927361A4 (en) | Treatment of diseases with multimeric peptides | |
EP3843791A4 (en) | Adeno-associated viral vectors for the treatment of best disease | |
IL268600A (en) | Methods for determining potency of adeno-associated virus preparations | |
GB201907305D0 (en) | Treatment of conditions | |
EP3823654C0 (en) | Methods for treatment of cancer using chikungunya-vsv chimeric virus |