BR112020014404A2 - Terapia gênica de vírus adenoassociado para deficiência de 21-hidroxilase - Google Patents
Terapia gênica de vírus adenoassociado para deficiência de 21-hidroxilase Download PDFInfo
- Publication number
- BR112020014404A2 BR112020014404A2 BR112020014404-5A BR112020014404A BR112020014404A2 BR 112020014404 A2 BR112020014404 A2 BR 112020014404A2 BR 112020014404 A BR112020014404 A BR 112020014404A BR 112020014404 A2 BR112020014404 A2 BR 112020014404A2
- Authority
- BR
- Brazil
- Prior art keywords
- adrenal
- vgc
- raav
- vector
- cyp21
- Prior art date
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Classifications
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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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/14—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced flavin or flavoprotein as one donor, and incorporation of one atom of oxygen (1.14.14)
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)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Mycology (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)
- Medicinal Preparation (AREA)
- Enzymes And Modification Thereof (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862618307P | 2018-01-17 | 2018-01-17 | |
| US62/618,307 | 2018-01-17 | ||
| US201862640311P | 2018-03-08 | 2018-03-08 | |
| US62/640,311 | 2018-03-08 | ||
| PCT/US2019/013991 WO2019143803A1 (en) | 2018-01-17 | 2019-01-17 | Adeno-associated virus gene therapy for 21-hydroxylase deficiency |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112020014404A2 true BR112020014404A2 (pt) | 2020-12-29 |
Family
ID=65494499
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112020014404-5A BR112020014404A2 (pt) | 2018-01-17 | 2019-01-17 | Terapia gênica de vírus adenoassociado para deficiência de 21-hidroxilase |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US12195767B2 (https=) |
| EP (1) | EP3740244B1 (https=) |
| JP (2) | JP7569216B2 (https=) |
| KR (1) | KR102892243B1 (https=) |
| CN (1) | CN111601620A (https=) |
| BR (1) | BR112020014404A2 (https=) |
| CA (1) | CA3088323A1 (https=) |
| WO (1) | WO2019143803A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2022198038A1 (en) | 2021-03-19 | 2022-09-22 | Adrenas Therapeutics, Inc. | Gene therapies for 21-hydroxylase deficiency |
| AU2022366947A1 (en) | 2021-10-12 | 2024-05-02 | Bridgebio Gene Therapy Llc | Methods and compositions for treating leukodystrophies |
| CN115976004B (zh) * | 2022-12-27 | 2024-07-09 | 天津科技大学 | 一种黄体酮17α-羟化酶突变体及其应用 |
| AU2024317892A1 (en) * | 2023-07-29 | 2025-08-28 | Limited Liability Company «Genetic Technologies» (Llc «Genetic Technologies») | Adeno-associated virus-based expression vectors |
Family Cites Families (15)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6136597A (en) | 1997-09-18 | 2000-10-24 | The Salk Institute For Biological Studies | RNA export element |
| WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| DK2573170T3 (en) | 2001-12-17 | 2018-04-09 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| 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 |
| US8865881B2 (en) * | 2011-02-22 | 2014-10-21 | California Institute Of Technology | Delivery of proteins using adeno-associated virus (AAV) vectors |
| IL297919A (en) | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| WO2015041718A1 (en) | 2013-09-20 | 2015-03-26 | The General Hospital Corporation | Uses of modified mullerian inhibiting substance (mis) proteins for the treatment of neurodegenerative diseases |
| EP3015550A1 (en) | 2014-10-30 | 2016-05-04 | Sanofi | 21-Hydroxylation of steroids |
| EP3054006A1 (en) | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Recombinant adeno-associated virus particle purification with multiple-step anion exchange chromatography |
| HUE069664T2 (hu) * | 2015-07-10 | 2025-04-28 | Univ Guelph | Madár-adenovírus 9 (FADV-9) vektorrendszer és kapcsolódó módszerek |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| WO2022198038A1 (en) | 2021-03-19 | 2022-09-22 | Adrenas Therapeutics, Inc. | Gene therapies for 21-hydroxylase deficiency |
-
2019
- 2019-01-17 CA CA3088323A patent/CA3088323A1/en active Pending
- 2019-01-17 EP EP19706059.3A patent/EP3740244B1/en active Active
- 2019-01-17 JP JP2020537156A patent/JP7569216B2/ja active Active
- 2019-01-17 KR KR1020207023328A patent/KR102892243B1/ko active Active
- 2019-01-17 BR BR112020014404-5A patent/BR112020014404A2/pt not_active Application Discontinuation
- 2019-01-17 US US16/962,552 patent/US12195767B2/en active Active
- 2019-01-17 CN CN201980008414.XA patent/CN111601620A/zh active Pending
- 2019-01-17 WO PCT/US2019/013991 patent/WO2019143803A1/en not_active Ceased
-
2023
- 2023-04-06 JP JP2023062403A patent/JP2023076698A/ja active Pending
-
2024
- 2024-11-13 US US18/946,854 patent/US20250304925A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3740244B1 (en) | 2025-05-21 |
| JP2021511020A (ja) | 2021-05-06 |
| EP3740244A1 (en) | 2020-11-25 |
| WO2019143803A1 (en) | 2019-07-25 |
| CN111601620A (zh) | 2020-08-28 |
| JP2023076698A (ja) | 2023-06-01 |
| US20250304925A1 (en) | 2025-10-02 |
| JP7569216B2 (ja) | 2024-10-17 |
| KR102892243B1 (ko) | 2025-11-27 |
| KR20200110376A (ko) | 2020-09-23 |
| CA3088323A1 (en) | 2019-07-25 |
| US20210277365A1 (en) | 2021-09-09 |
| US12195767B2 (en) | 2025-01-14 |
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| B350 | Update of information on the portal [chapter 15.35 patent gazette] | ||
| B06W | Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette] | ||
| B11B | Dismissal acc. art. 36, par 1 of ipl - no reply within 90 days to fullfil the necessary requirements |