MX2020006764A - Una proteína de la cápside de raav modificada para terapia génica. - Google Patents
Una proteína de la cápside de raav modificada para terapia génica.Info
- Publication number
- MX2020006764A MX2020006764A MX2020006764A MX2020006764A MX2020006764A MX 2020006764 A MX2020006764 A MX 2020006764A MX 2020006764 A MX2020006764 A MX 2020006764A MX 2020006764 A MX2020006764 A MX 2020006764A MX 2020006764 A MX2020006764 A MX 2020006764A
- Authority
- MX
- Mexico
- Prior art keywords
- gene therapy
- capsid protein
- raav capsid
- modified raav
- virions
- 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
- A61K48/0008—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
- A61K48/0025—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 non-active part clearly interacts with the delivered nucleic acid
-
- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
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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
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14145—Special targeting system for viral vectors
-
- 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)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Public Health (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Epidemiology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Rheumatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Dermatology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
El invento se refiere a viriones de virus adenoasociados recombinantes (en inglés rAAV) para terapia génica, en los que los viriones rAAV comprenden una nueva proteína de la cápside. En particular, el invento se refiere al uso de tales viriones en terapia génica para el tratamiento de una enfermedad artrítica, como, por ejemplo, artritis reumatoide, o síntomas de la misma, preferiblemente por administración intraarticular.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP18152133 | 2018-01-17 | ||
PCT/EP2019/051128 WO2019141765A1 (en) | 2018-01-17 | 2019-01-17 | A modified raav capsid protein for gene therapy |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2020006764A true MX2020006764A (es) | 2021-01-08 |
Family
ID=61022137
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2020006764A MX2020006764A (es) | 2018-01-17 | 2019-01-17 | Una proteína de la cápside de raav modificada para terapia génica. |
Country Status (24)
Country | Link |
---|---|
US (1) | US20200354744A1 (es) |
EP (1) | EP3740222B1 (es) |
JP (1) | JP7389040B2 (es) |
KR (1) | KR20200135768A (es) |
CN (1) | CN112004544A (es) |
AU (1) | AU2019209595B2 (es) |
BR (1) | BR112020014625A2 (es) |
CA (1) | CA3087910A1 (es) |
DK (1) | DK3740222T3 (es) |
EA (1) | EA202091712A1 (es) |
ES (1) | ES2957622T3 (es) |
FI (1) | FI3740222T3 (es) |
HR (1) | HRP20231126T1 (es) |
HU (1) | HUE062774T2 (es) |
IL (1) | IL275838A (es) |
LT (1) | LT3740222T (es) |
MX (1) | MX2020006764A (es) |
PH (1) | PH12020551096A1 (es) |
PL (1) | PL3740222T3 (es) |
PT (1) | PT3740222T (es) |
RS (1) | RS64499B1 (es) |
SG (1) | SG11202006056RA (es) |
SI (1) | SI3740222T1 (es) |
WO (1) | WO2019141765A1 (es) |
Families Citing this family (8)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP3831842A1 (en) | 2015-09-28 | 2021-06-09 | The University of North Carolina at Chapel Hill | Methods and compositions for antibody-evading virus vectors |
EP3645021A4 (en) * | 2017-06-30 | 2021-04-21 | Intima Bioscience, Inc. | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY |
EP3774854A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
CN110437317B (zh) * | 2019-01-30 | 2023-05-02 | 上海科技大学 | 具有变异衣壳蛋白的腺相关病毒及其用途 |
TW202102525A (zh) | 2019-03-21 | 2021-01-16 | 美商史崔德生物公司 | 重組腺相關病毒載體 |
MX2022000551A (es) * | 2019-07-15 | 2022-05-18 | Meiragtx Uk Ii Ltd | Proteínas de la cápside de aav modificadas para el tratamiento de una enfermedad artrítica. |
MX2022004352A (es) | 2019-10-17 | 2022-07-19 | Stridebio Inc | Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c. |
CN110950934B (zh) * | 2019-12-31 | 2022-11-04 | 复旦大学 | 一种腺相关病毒衣壳蛋白、载体及其构建方法与应用 |
Family Cites Families (19)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA2467959C (en) | 2001-11-09 | 2009-03-10 | Robert M. Kotin | Production of adeno-associated virus in insect cells |
US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
WO2005021768A1 (en) * | 2003-09-01 | 2005-03-10 | Academisch Medisch Centrum | Aav vectors for in vivo gene therapy of rheumatoid arthritis |
NZ545656A (en) * | 2003-09-01 | 2009-02-28 | Amc Amsterdam | AAV vectors for in vivo gene therapy of rheumatoid arthritis |
AU2010201278B2 (en) * | 2003-09-01 | 2012-11-15 | Academisch Medisch Centrum | AAV vectors for in vivo gene therapy of rheumatoid arthritis |
CA2622233C (en) | 2005-10-20 | 2015-01-20 | Amsterdam Molecular Therapeutics (Amt) B.V. | Improved aav vectors produced in insect cells |
WO2007148971A2 (en) | 2006-06-21 | 2007-12-27 | Amsterdam Molecular Therapeutics B.V. | Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells |
US8945918B2 (en) | 2006-08-24 | 2015-02-03 | Virovek, Inc. | Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor |
DK3093345T3 (da) | 2007-07-26 | 2019-06-24 | Uniqure Ip Bv | Baculovirusvektorer omfattende gentagne kodende sekvenser med differentielle foretrukne kodoner |
ES2644880T3 (es) | 2008-02-19 | 2017-11-30 | Uniqure Ip B.V. | Optimización de expresión de proteínas Rep y Cap parvovirales en las células de insecto |
EP2297185A1 (en) | 2008-06-17 | 2011-03-23 | Amsterdam Molecular Therapeutics (AMT) B.V. | Parvoviral capsid with incorporated gly-ala repeat region |
US9228174B2 (en) | 2010-03-11 | 2016-01-05 | Uniqure Ip B.V. | Mutated rep encoding sequences for use in AAV production |
DK2744895T3 (en) | 2011-09-08 | 2016-01-04 | Uniqure Ip Bv | REMOVAL OF INFECTED VIRA FROM AAV PREPARATIONS |
WO2013174760A1 (en) * | 2012-05-25 | 2013-11-28 | Deutsches Krebsforschungszentrum | Optimized aav-vectors for high transduction rates in dendritic cells |
DE102014207498A1 (de) * | 2014-04-17 | 2015-10-22 | Universitätsklinikum Hamburg-Eppendorf | Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark |
CN104107438A (zh) * | 2014-05-29 | 2014-10-22 | 中国人民解放军军事医学科学院基础医学研究所 | Spry2在制备预防和治疗类风湿性关节炎药物中的应用 |
US20170304466A1 (en) * | 2014-10-06 | 2017-10-26 | Arthrogen B.V. | AAV-Based Gene Therapy |
CA2967468A1 (en) * | 2014-12-16 | 2016-06-23 | Board Of Regents Of The University Of Nebraska | Gene therapy for juvenile batten disease |
MX2022000551A (es) * | 2019-07-15 | 2022-05-18 | Meiragtx Uk Ii Ltd | Proteínas de la cápside de aav modificadas para el tratamiento de una enfermedad artrítica. |
-
2019
- 2019-01-17 PL PL19701080.4T patent/PL3740222T3/pl unknown
- 2019-01-17 AU AU2019209595A patent/AU2019209595B2/en active Active
- 2019-01-17 ES ES19701080T patent/ES2957622T3/es active Active
- 2019-01-17 JP JP2020539801A patent/JP7389040B2/ja active Active
- 2019-01-17 EP EP19701080.4A patent/EP3740222B1/en active Active
- 2019-01-17 BR BR112020014625-0A patent/BR112020014625A2/pt unknown
- 2019-01-17 SI SI201930613T patent/SI3740222T1/sl unknown
- 2019-01-17 CA CA3087910A patent/CA3087910A1/en active Pending
- 2019-01-17 FI FIEP19701080.4T patent/FI3740222T3/fi active
- 2019-01-17 HR HRP20231126TT patent/HRP20231126T1/hr unknown
- 2019-01-17 SG SG11202006056RA patent/SG11202006056RA/en unknown
- 2019-01-17 PT PT197010804T patent/PT3740222T/pt unknown
- 2019-01-17 HU HUE19701080A patent/HUE062774T2/hu unknown
- 2019-01-17 LT LTEPPCT/EP2019/051128T patent/LT3740222T/lt unknown
- 2019-01-17 US US16/962,748 patent/US20200354744A1/en active Pending
- 2019-01-17 CN CN201980020100.1A patent/CN112004544A/zh active Pending
- 2019-01-17 DK DK19701080.4T patent/DK3740222T3/da active
- 2019-01-17 WO PCT/EP2019/051128 patent/WO2019141765A1/en active Application Filing
- 2019-01-17 EA EA202091712A patent/EA202091712A1/ru unknown
- 2019-01-17 MX MX2020006764A patent/MX2020006764A/es unknown
- 2019-01-17 RS RS20230730A patent/RS64499B1/sr unknown
- 2019-01-17 KR KR1020207023174A patent/KR20200135768A/ko active Search and Examination
-
2020
- 2020-07-05 IL IL275838A patent/IL275838A/en unknown
- 2020-07-17 PH PH12020551096A patent/PH12020551096A1/en unknown
Also Published As
Publication number | Publication date |
---|---|
NZ767106A (en) | 2023-10-27 |
PH12020551096A1 (en) | 2021-06-21 |
PL3740222T3 (pl) | 2023-12-04 |
BR112020014625A2 (pt) | 2020-12-08 |
CN112004544A (zh) | 2020-11-27 |
FI3740222T3 (fi) | 2023-07-26 |
SI3740222T1 (sl) | 2023-11-30 |
JP2021510528A (ja) | 2021-04-30 |
HUE062774T2 (hu) | 2023-12-28 |
RS64499B1 (sr) | 2023-09-29 |
HRP20231126T1 (hr) | 2024-01-05 |
DK3740222T3 (da) | 2023-07-31 |
JP7389040B2 (ja) | 2023-11-29 |
AU2019209595A1 (en) | 2020-09-03 |
IL275838A (en) | 2020-08-31 |
CA3087910A1 (en) | 2019-07-25 |
ES2957622T3 (es) | 2024-01-23 |
EA202091712A1 (ru) | 2021-02-16 |
SG11202006056RA (en) | 2020-07-29 |
EP3740222A1 (en) | 2020-11-25 |
KR20200135768A (ko) | 2020-12-03 |
LT3740222T (lt) | 2023-09-11 |
US20200354744A1 (en) | 2020-11-12 |
EP3740222B1 (en) | 2023-06-28 |
WO2019141765A1 (en) | 2019-07-25 |
PT3740222T (pt) | 2023-07-11 |
AU2019209595B2 (en) | 2023-04-27 |
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