CL2022001260A1 - Tratamiento del angioedema hereditario con vectores de terapia de genes específicos del hígado - Google Patents
Tratamiento del angioedema hereditario con vectores de terapia de genes específicos del hígadoInfo
- Publication number
- CL2022001260A1 CL2022001260A1 CL2022001260A CL2022001260A CL2022001260A1 CL 2022001260 A1 CL2022001260 A1 CL 2022001260A1 CL 2022001260 A CL2022001260 A CL 2022001260A CL 2022001260 A CL2022001260 A CL 2022001260A CL 2022001260 A1 CL2022001260 A1 CL 2022001260A1
- Authority
- CL
- Chile
- Prior art keywords
- liver
- treatment
- gene therapy
- specific gene
- therapy vectors
- Prior art date
Links
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/005—Enzyme inhibitors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/10—Antioedematous agents; Diuretics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/81—Protease inhibitors
- C07K14/8107—Endopeptidase (E.C. 3.4.21-99) inhibitors
- C07K14/811—Serine protease (E.C. 3.4.21) inhibitors
- C07K14/8121—Serpins
-
- 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
- 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
-
- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/14011—Baculoviridae
- C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
- C12N2710/14141—Use of virus, viral particle or viral elements as a vector
- C12N2710/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
- C12N2750/14122—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
- 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
- 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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Diabetes (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Hematology (AREA)
- Immunology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
Aquí se proporcionan composiciones y métodos para tratar una deficiencia del inhibidor de la esterasa C1 normalizando los niveles de la proteína inhibidora de la esterasa C1 en un sujeto que tiene HAE.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962935359P | 2019-11-14 | 2019-11-14 | |
US202063016365P | 2020-04-28 | 2020-04-28 |
Publications (1)
Publication Number | Publication Date |
---|---|
CL2022001260A1 true CL2022001260A1 (es) | 2023-03-10 |
Family
ID=73834586
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2022001260A CL2022001260A1 (es) | 2019-11-14 | 2022-05-13 | Tratamiento del angioedema hereditario con vectores de terapia de genes específicos del hígado |
Country Status (14)
Country | Link |
---|---|
US (1) | US20230340078A1 (es) |
EP (1) | EP4058475A1 (es) |
JP (1) | JP2023503850A (es) |
KR (1) | KR20220098210A (es) |
CN (1) | CN114829391A (es) |
AU (1) | AU2020384294A1 (es) |
BR (1) | BR112022009279A2 (es) |
CA (1) | CA3161154A1 (es) |
CL (1) | CL2022001260A1 (es) |
CO (1) | CO2022005641A2 (es) |
IL (1) | IL292717A (es) |
MX (1) | MX2022005869A (es) |
TW (1) | TW202128733A (es) |
WO (1) | WO2021097157A1 (es) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
KR20230078805A (ko) | 2020-11-02 | 2023-06-02 | 바이오마린 파머수티컬 인크. | 아데노-연관 바이러스의 농축 공정 |
AU2022292164A1 (en) * | 2021-06-17 | 2024-02-01 | Meiragtx Uk Ii Limited | Aav manufacturing methods |
AR127217A1 (es) | 2021-10-01 | 2023-12-27 | Biomarin Pharm Inc | Tratamiento de angioedema hereditario con vectores de genoterapia de aav y formulaciones terapéuticas |
Family Cites Families (14)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4745051A (en) | 1983-05-27 | 1988-05-17 | The Texas A&M University System | Method for producing a recombinant baculovirus expression vector |
EP0127839B1 (en) | 1983-05-27 | 1992-07-15 | THE TEXAS A&M UNIVERSITY SYSTEM | Method for producing a recombinant baculovirus expression vector |
ZA848495B (en) | 1984-01-31 | 1985-09-25 | Idaho Res Found | Production of polypeptides in insect cells |
US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
WO2003033710A1 (fr) | 2001-10-16 | 2003-04-24 | National Institute Of Advanced Industrial Science And Technology | Nouvelle n-acetylglucosamine transferase, acide nucleique codant cette enzyme, anticorps dirige contre cette enzyme et utilisation de l'enzyme pour diagnostiquer un cancer ou une tumeur |
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 |
EP2338900B1 (en) | 2001-11-13 | 2014-01-01 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) Cy.5 sequences, vectors containing the same and uses thereof |
WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
US7943379B2 (en) | 2008-04-30 | 2011-05-17 | Nationwide Children's Hospital, Inc. | Production of rAAV in vero cells using particular adenovirus helpers |
RU2653444C2 (ru) | 2012-02-17 | 2018-05-08 | Дзе Чилдрен'З Хоспитал Оф Филадельфия | Композиции вектора aav и способы переноса генов в клетки, органы и ткани |
PE20160188A1 (es) * | 2013-07-22 | 2016-04-27 | Philadelphia Children Hospital | Variantes aav y composiciones, metodos y usos para transferencia de gen a celulas, organos y tejidos |
AU2016267687B2 (en) * | 2015-05-28 | 2022-04-07 | Cornell University | Adeno-associated virus mediated delivery of C1EI as a therapy for angioedema |
SG11201811603WA (en) | 2016-07-26 | 2019-02-27 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
WO2019143272A1 (en) * | 2018-01-16 | 2019-07-25 | Cls Therapeutics Limited | Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (dnase) activity |
-
2020
- 2020-11-13 KR KR1020227019436A patent/KR20220098210A/ko active Search and Examination
- 2020-11-13 CA CA3161154A patent/CA3161154A1/en active Pending
- 2020-11-13 CN CN202080087888.0A patent/CN114829391A/zh active Pending
- 2020-11-13 WO PCT/US2020/060337 patent/WO2021097157A1/en active Application Filing
- 2020-11-13 US US17/777,690 patent/US20230340078A1/en active Pending
- 2020-11-13 MX MX2022005869A patent/MX2022005869A/es unknown
- 2020-11-13 EP EP20824369.1A patent/EP4058475A1/en active Pending
- 2020-11-13 BR BR112022009279A patent/BR112022009279A2/pt unknown
- 2020-11-13 TW TW109139830A patent/TW202128733A/zh unknown
- 2020-11-13 JP JP2022527987A patent/JP2023503850A/ja active Pending
- 2020-11-13 AU AU2020384294A patent/AU2020384294A1/en active Pending
-
2022
- 2022-04-29 CO CONC2022/0005641A patent/CO2022005641A2/es unknown
- 2022-05-03 IL IL292717A patent/IL292717A/en unknown
- 2022-05-13 CL CL2022001260A patent/CL2022001260A1/es unknown
Also Published As
Publication number | Publication date |
---|---|
TW202128733A (zh) | 2021-08-01 |
EP4058475A1 (en) | 2022-09-21 |
CA3161154A1 (en) | 2021-05-20 |
AU2020384294A1 (en) | 2022-06-02 |
BR112022009279A2 (pt) | 2022-09-06 |
JP2023503850A (ja) | 2023-02-01 |
CO2022005641A2 (es) | 2022-05-20 |
IL292717A (en) | 2022-07-01 |
CN114829391A (zh) | 2022-07-29 |
MX2022005869A (es) | 2022-06-14 |
WO2021097157A1 (en) | 2021-05-20 |
US20230340078A1 (en) | 2023-10-26 |
KR20220098210A (ko) | 2022-07-11 |
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