MX2021008832A - Composiciones y metodos para la administracion de polipeptidos cftr. - Google Patents

Composiciones y metodos para la administracion de polipeptidos cftr.

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Publication number
MX2021008832A
MX2021008832A MX2021008832A MX2021008832A MX2021008832A MX 2021008832 A MX2021008832 A MX 2021008832A MX 2021008832 A MX2021008832 A MX 2021008832A MX 2021008832 A MX2021008832 A MX 2021008832A MX 2021008832 A MX2021008832 A MX 2021008832A
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Mexico
Prior art keywords
compositions
methods
nucleic acids
recombinant nucleic
viruses
Prior art date
Application number
MX2021008832A
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English (en)
Inventor
Suma Krishnan
Trevor Parry
Pooja Agarwal
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Krystal Biotech Inc
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Publication date
Application filed by Krystal Biotech Inc filed Critical Krystal Biotech Inc
Publication of MX2021008832A publication Critical patent/MX2021008832A/es

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/007Pulmonary tract; Aromatherapy
    • A61K9/0073Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712Cystic fibrosis
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    • A61K9/0073Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0075Sprays 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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    • A61K9/0078Sprays 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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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.
MX2021008832A 2019-02-08 2020-02-07 Composiciones y metodos para la administracion de polipeptidos cftr. MX2021008832A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962802871P 2019-02-08 2019-02-08
PCT/US2020/017191 WO2020163703A1 (en) 2019-02-08 2020-02-07 Compositions and methods for delivering cftr polypeptides

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MX2021008832A true MX2021008832A (es) 2021-09-08

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MX2021008832A MX2021008832A (es) 2019-02-08 2020-02-07 Composiciones y metodos para la administracion de polipeptidos cftr.

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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)

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