CO2021009575A2 - Composiciones y métodos para la administración de polipéptidos cftr - Google Patents

Composiciones y métodos para la administración de polipéptidos cftr

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Publication number
CO2021009575A2
CO2021009575A2 CONC2021/0009575A CO2021009575A CO2021009575A2 CO 2021009575 A2 CO2021009575 A2 CO 2021009575A2 CO 2021009575 A CO2021009575 A CO 2021009575A CO 2021009575 A2 CO2021009575 A2 CO 2021009575A2
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Colombia
Prior art keywords
compositions
methods
nucleic acids
cftr
administration
Prior art date
Application number
CONC2021/0009575A
Other languages
English (en)
Inventor
Suma Krishnan
Trevor Parry
Pooja Agarwal
Original Assignee
Krystal Biotech Inc
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Publication date
Application filed by Krystal Biotech Inc filed Critical Krystal Biotech Inc
Publication of CO2021009575A2 publication Critical patent/CO2021009575A2/es

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    • 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
    • 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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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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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    • 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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    • 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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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.
CONC2021/0009575A 2019-02-08 2021-07-22 Composiciones y métodos para la administración de polipéptidos cftr CO2021009575A2 (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

Publications (1)

Publication Number Publication Date
CO2021009575A2 true CO2021009575A2 (es) 2021-08-09

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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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JP2022519734A (ja) * 2019-02-08 2022-03-24 クリスタル バイオテック インコーポレイテッド Ｃｆｔｒポリペプチドを送達するための組成物及び方法
JP2024513826A (ja) 2021-04-02 2024-03-27 クリスタル バイオテック インコーポレイテッド がん治療のためのウイルスベクター
WO2024052734A2 (en) * 2022-07-13 2024-03-14 Intergalactic Therapeutics, Inc. Respiratory vectors and uses thereof

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CA3127133A1 (en) 2020-08-13
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