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PE20180675A1 - Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos - Google Patents

Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos

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Publication number
PE20180675A1
PE20180675A1 PE2017002835A PE2017002835A PE20180675A1 PE 20180675 A1 PE20180675 A1 PE 20180675A1 PE 2017002835 A PE2017002835 A PE 2017002835A PE 2017002835 A PE2017002835 A PE 2017002835A PE 20180675 A1 PE20180675 A1 PE 20180675A1
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PE
Peru
Prior art keywords
organs
tissues
cells
methods
genes
Prior art date
Application number
PE2017002835A
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English (en)
Inventor
Katherine A High
Xavier Anguela
Original Assignee
Childrens Hospital Philadelphia
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Childrens Hospital Philadelphia filed Critical Childrens Hospital Philadelphia
Publication of PE20180675A1 publication Critical patent/PE20180675A1/es

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    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • 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
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
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    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • 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
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    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
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    • 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

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Abstract

La invencion se refiere a una secuencia que codifica el Factor IX modificado, el casete de expresion, los vectores tales como vectores virales (por ejemplo, lenti- o adenoasociados virales), y metodos y usos de transferencia genetica. En particular, se refiere a dirigir el acido nucleico del Factor IX a las celulas, los tejidos u organos para la expresion (transcripcion) del Factor IX
PE2017002835A 2015-06-23 2016-06-23 Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos PE20180675A1 (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562183599P 2015-06-23 2015-06-23
US201662315453P 2016-03-30 2016-03-30
US201662338315P 2016-05-18 2016-05-18
US201662348781P 2016-06-10 2016-06-10
US201662349572P 2016-06-13 2016-06-13

Publications (1)

Publication Number Publication Date
PE20180675A1 true PE20180675A1 (es) 2018-04-19

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ID=57586246

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PE2017002835A PE20180675A1 (es) 2015-06-23 2016-06-23 Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos

Country Status (25)

Country Link
US (6) US10799566B2 (es)
EP (2) EP4378487A3 (es)
JP (3) JP6831779B2 (es)
KR (2) KR102415896B1 (es)
CN (1) CN108138159A (es)
AU (2) AU2016282781A1 (es)
CA (1) CA2990193A1 (es)
CO (1) CO2018000134A2 (es)
DK (1) DK3313991T3 (es)
ES (1) ES2989774T3 (es)
FI (2) FI3313991T3 (es)
FR (1) FR25C1002I1 (es)
HR (1) HRP20241180T1 (es)
HU (2) HUE068603T2 (es)
IL (2) IL256517B2 (es)
MX (2) MX390848B (es)
NO (1) NO2025003I1 (es)
PE (1) PE20180675A1 (es)
PH (1) PH12017502424A1 (es)
PL (1) PL3313991T3 (es)
PT (1) PT3313991T (es)
SA (1) SA517390590B1 (es)
SG (1) SG10202100131WA (es)
SI (1) SI3313991T1 (es)
WO (1) WO2016210170A1 (es)

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