BR112021020708A2 - Composições e métodos para tratamento de fibrose cística - Google Patents
Composições e métodos para tratamento de fibrose císticaInfo
- Publication number
- BR112021020708A2 BR112021020708A2 BR112021020708A BR112021020708A BR112021020708A2 BR 112021020708 A2 BR112021020708 A2 BR 112021020708A2 BR 112021020708 A BR112021020708 A BR 112021020708A BR 112021020708 A BR112021020708 A BR 112021020708A BR 112021020708 A2 BR112021020708 A2 BR 112021020708A2
- Authority
- BR
- Brazil
- Prior art keywords
- methods
- cystic fibrosis
- compositions
- treating cystic
- treating
- Prior art date
Links
Classifications
-
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in 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
-
- 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
-
- 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
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
-
- 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
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962833972P | 2019-04-15 | 2019-04-15 | |
| US201962926308P | 2019-10-25 | 2019-10-25 | |
| US202062967214P | 2020-01-29 | 2020-01-29 | |
| PCT/US2020/028264 WO2020214668A1 (en) | 2019-04-15 | 2020-04-15 | Compositions and methods for treatment of cystic fibrosis |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112021020708A2 true BR112021020708A2 (pt) | 2022-03-15 |
Family
ID=70847479
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112021020708A BR112021020708A2 (pt) | 2019-04-15 | 2020-04-15 | Composições e métodos para tratamento de fibrose cística |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20220241436A1 (https=) |
| EP (1) | EP3955971A1 (https=) |
| JP (1) | JP7731801B2 (https=) |
| KR (1) | KR20220044899A (https=) |
| CN (1) | CN114641318A (https=) |
| AU (1) | AU2020260076A1 (https=) |
| BR (1) | BR112021020708A2 (https=) |
| CA (1) | CA3137015A1 (https=) |
| CL (1) | CL2021002702A1 (https=) |
| IL (1) | IL287260A (https=) |
| MX (1) | MX2021012681A (https=) |
| PH (1) | PH12021552610A1 (https=) |
| SG (1) | SG11202111334SA (https=) |
| WO (1) | WO2020214668A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2017139381A1 (en) | 2016-02-08 | 2017-08-17 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| CA3198936A1 (en) | 2016-03-07 | 2017-09-14 | University Of Iowa Research Foundation | Aav-mediated expression using a synthetic promoter and enhancer |
| US12173305B2 (en) | 2016-05-26 | 2024-12-24 | University Of Iowa Research Foundation | cis and trans requirements for terminal resolution of human bocavirus 1 |
| US11142775B2 (en) | 2017-01-13 | 2021-10-12 | University Of Iowa Research Foundation | Bocaparvovirus small noncoding RNA and uses thereof |
| EP3955970A1 (en) * | 2019-04-15 | 2022-02-23 | University Of Iowa Research Foundation | Methods and compositions for transgene expression |
| US20230242941A1 (en) * | 2020-06-30 | 2023-08-03 | Eric Yuen | Methods and compositions for administering recombinant viral vectors |
| US20240115738A1 (en) | 2021-04-15 | 2024-04-11 | Spirovant Sciences, Inc. | Methods and compositions for treatment of cystic fibrosis |
| WO2026072721A1 (en) * | 2024-09-25 | 2026-04-02 | Carbon Biosciences, Inc. | Parvovirus compositions and methods for gene therapy |
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 |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| US5656785A (en) | 1995-08-07 | 1997-08-12 | The Charles Stark Draper Laboratory, Inc. | Micromechanical contact load force sensor for sensing magnitude and distribution of loads and tool employing micromechanical contact load force sensor |
| AU5445998A (en) | 1996-11-19 | 1998-06-10 | Surgx Corporation | A transient voltage protection device and method of making same |
| AU741605B2 (en) | 1996-12-18 | 2001-12-06 | Targeted Genetics Corporation | AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors |
| JP2007524379A (ja) | 2003-03-31 | 2007-08-30 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | 上皮ナトリウムチャンネル関連障害のＰｈａｒｍｉｃｏ−遺伝子治療 |
| US9233131B2 (en) * | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| WO2005056762A2 (en) * | 2003-12-05 | 2005-06-23 | University Of Iowa Research Foundation | Truncated cmv promoters and vectors containing same |
| KR20210122917A (ko) * | 2013-03-14 | 2021-10-12 | 샤이어 휴먼 지네틱 테라피즈 인크. | Cftr mrna 조성물 및 관련 방법 및 사용 |
| AU2014251099B2 (en) * | 2013-04-08 | 2019-01-17 | The University Of Kansas | Chimeric adeno-associated virus/ bocavirus parvovirus vector |
| US11000597B2 (en) * | 2015-01-21 | 2021-05-11 | University Of Florida Research Foundation, Incorporated | Engineered receptor/ligand system for delivery of therapeutic agents |
| CA3198936A1 (en) * | 2016-03-07 | 2017-09-14 | University Of Iowa Research Foundation | Aav-mediated expression using a synthetic promoter and enhancer |
| US10550405B2 (en) * | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| EP3955970A1 (en) * | 2019-04-15 | 2022-02-23 | University Of Iowa Research Foundation | Methods and compositions for transgene expression |
-
2020
- 2020-04-15 US US17/603,831 patent/US20220241436A1/en not_active Abandoned
- 2020-04-15 JP JP2021561742A patent/JP7731801B2/ja active Active
- 2020-04-15 WO PCT/US2020/028264 patent/WO2020214668A1/en not_active Ceased
- 2020-04-15 PH PH1/2021/552610A patent/PH12021552610A1/en unknown
- 2020-04-15 KR KR1020217037210A patent/KR20220044899A/ko not_active Ceased
- 2020-04-15 MX MX2021012681A patent/MX2021012681A/es unknown
- 2020-04-15 SG SG11202111334SA patent/SG11202111334SA/en unknown
- 2020-04-15 AU AU2020260076A patent/AU2020260076A1/en active Pending
- 2020-04-15 BR BR112021020708A patent/BR112021020708A2/pt unknown
- 2020-04-15 CN CN202080043595.2A patent/CN114641318A/zh active Pending
- 2020-04-15 CA CA3137015A patent/CA3137015A1/en active Pending
- 2020-04-15 EP EP20728248.4A patent/EP3955971A1/en active Pending
-
2021
- 2021-10-14 IL IL287260A patent/IL287260A/en unknown
- 2021-10-15 CL CL2021002702A patent/CL2021002702A1/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| AU2020260076A1 (en) | 2021-12-09 |
| JP2022529457A (ja) | 2022-06-22 |
| WO2020214668A1 (en) | 2020-10-22 |
| PH12021552610A1 (en) | 2022-06-27 |
| CN114641318A (zh) | 2022-06-17 |
| CL2021002702A1 (es) | 2022-11-11 |
| SG11202111334SA (en) | 2021-11-29 |
| CA3137015A1 (en) | 2020-10-22 |
| IL287260A (en) | 2021-12-01 |
| JP7731801B2 (ja) | 2025-09-01 |
| KR20220044899A (ko) | 2022-04-12 |
| US20220241436A1 (en) | 2022-08-04 |
| MX2021012681A (es) | 2022-03-25 |
| EP3955971A1 (en) | 2022-02-23 |
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