CL2021002078A1 - Composiciones y métodos para la administración de polipéptidos cftr. - Google Patents
Composiciones y métodos para la administración de polipéptidos cftr.Info
- Publication number
- CL2021002078A1 CL2021002078A1 CL2021002078A CL2021002078A CL2021002078A1 CL 2021002078 A1 CL2021002078 A1 CL 2021002078A1 CL 2021002078 A CL2021002078 A CL 2021002078A CL 2021002078 A CL2021002078 A CL 2021002078A CL 2021002078 A1 CL2021002078 A1 CL 2021002078A1
- Authority
- CL
- Chile
- Prior art keywords
- compositions
- methods
- nucleic acids
- cftr
- administration
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
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- 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
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- 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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4712—Cystic fibrosis
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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
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- 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
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- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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- 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
- A61K9/007—Pulmonary tract; Aromatherapy
- A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
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- 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
- A61K9/007—Pulmonary tract; Aromatherapy
- A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
- A61K9/0075—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a dry powder inhaler [DPI], e.g. comprising micronized drug mixed with lactose carrier particles
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- 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
- A61K9/007—Pulmonary tract; Aromatherapy
- A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
- A61K9/0078—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16041—Use of virus, viral particle or viral elements as a vector
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16041—Use of virus, viral particle or viral elements as a vector
- C12N2710/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16071—Demonstrated in vivo effect
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
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- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16511—Roseolovirus, e.g. human herpesvirus 6, 7
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- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
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- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
- C12N2710/16621—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
- C12N2710/16632—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
- C12N2710/16641—Use of virus, viral particle or viral elements as a vector
- C12N2710/16643—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
- C12N2710/16671—Demonstrated in vivo effect
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16711—Varicellovirus, e.g. human herpesvirus 3, Varicella Zoster, pseudorabies
Abstract
La presente divulgación proporciona ácidos nucleicos recombinantes que comprenden uno o más polinucleótidos que codifican un polipéptido del regulador de conductancia transmembrana de fibrosis quística (CFTR) (por ejemplo, un polinucleótido CFTR humano); virus que comprenden los ácidos nucleicos recombinantes; composiciones y formulaciones que comprenden los virus y/o ácidos nucleicos recombinantes; métodos para utilizarlos (por ejemplo, para el tratamiento de una enfermedad pulmonar crónica, tal como fibrosis quística); y artículos de elaboración o kits de estos.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962802871P | 2019-02-08 | 2019-02-08 |
Publications (1)
Publication Number | Publication Date |
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CL2021002078A1 true CL2021002078A1 (es) | 2022-02-04 |
Family
ID=69740894
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2021002078A CL2021002078A1 (es) | 2019-02-08 | 2021-08-05 | Composiciones y métodos para la administración de polipéptidos cftr. |
Country Status (14)
Country | Link |
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US (3) | US10829529B2 (es) |
EP (1) | EP3921334A1 (es) |
JP (1) | JP2022519734A (es) |
KR (1) | KR20210124277A (es) |
CN (1) | CN113454105A (es) |
AU (1) | AU2020219343A1 (es) |
BR (1) | BR112021014400A2 (es) |
CA (1) | CA3127133A1 (es) |
CL (1) | CL2021002078A1 (es) |
CO (1) | CO2021009575A2 (es) |
MX (1) | MX2021008832A (es) |
SG (1) | SG11202107969SA (es) |
WO (1) | WO2020163703A1 (es) |
ZA (1) | ZA202205420B (es) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU2020219343A1 (en) * | 2019-02-08 | 2021-08-19 | Krystal Biotech, Inc. | Compositions and methods for delivering CFTR polypeptides |
BR112023020209A2 (pt) | 2021-04-02 | 2023-12-19 | Krystal Biotech Inc | Genoma do vírus do herpes recombinante, vírus do herpes, composição farmacêutica, uso do vírus do herpes ou da composição farmacêutica, método para expressar, realçar, aumentar, ampliar e/ou suplementar os níveis de um polipeptídeo imunomodulatório, método para prover alívio profilático, paliativo ou terapêutico, e, método para tratar câncer |
WO2024052734A2 (en) * | 2022-07-13 | 2024-03-14 | Intergalactic Therapeutics, Inc. | Respiratory vectors and uses thereof |
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AU2019292580A1 (en) | 2018-06-29 | 2021-02-04 | Krystal Biotech, Inc. | Compositions and methods for antibody delivery |
WO2020068862A1 (en) | 2018-09-24 | 2020-04-02 | Krystal Biotech, Inc. | Compositions and methods for the treatment of netherton syndrome |
AU2020219343A1 (en) * | 2019-02-08 | 2021-08-19 | Krystal Biotech, Inc. | Compositions and methods for delivering CFTR polypeptides |
-
2020
- 2020-02-07 AU AU2020219343A patent/AU2020219343A1/en active Pending
- 2020-02-07 CN CN202080013119.6A patent/CN113454105A/zh active Pending
- 2020-02-07 KR KR1020217026522A patent/KR20210124277A/ko unknown
- 2020-02-07 JP JP2021546328A patent/JP2022519734A/ja active Pending
- 2020-02-07 MX MX2021008832A patent/MX2021008832A/es unknown
- 2020-02-07 CA CA3127133A patent/CA3127133A1/en active Pending
- 2020-02-07 US US16/784,599 patent/US10829529B2/en active Active
- 2020-02-07 EP EP20709107.5A patent/EP3921334A1/en active Pending
- 2020-02-07 SG SG11202107969SA patent/SG11202107969SA/en unknown
- 2020-02-07 WO PCT/US2020/017191 patent/WO2020163703A1/en active Application Filing
- 2020-02-07 BR BR112021014400-5A patent/BR112021014400A2/pt unknown
- 2020-11-06 US US17/092,119 patent/US20210087245A1/en active Pending
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2021
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- 2021-07-22 CO CONC2021/0009575A patent/CO2021009575A2/es unknown
- 2021-08-05 CL CL2021002078A patent/CL2021002078A1/es unknown
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2022
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US20200254060A1 (en) | 2020-08-13 |
KR20210124277A (ko) | 2021-10-14 |
CA3127133A1 (en) | 2020-08-13 |
BR112021014400A2 (pt) | 2021-09-21 |
US10829529B2 (en) | 2020-11-10 |
AU2020219343A1 (en) | 2021-08-19 |
US20210087245A1 (en) | 2021-03-25 |
US20210317174A1 (en) | 2021-10-14 |
MX2021008832A (es) | 2021-09-08 |
CN113454105A (zh) | 2021-09-28 |
CO2021009575A2 (es) | 2021-08-09 |
JP2022519734A (ja) | 2022-03-24 |
ZA202205420B (en) | 2022-09-28 |
SG11202107969SA (en) | 2021-08-30 |
WO2020163703A1 (en) | 2020-08-13 |
EP3921334A1 (en) | 2021-12-15 |
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