MX2021001901A - Factor ix encoding nucleotides. - Google Patents

Factor ix encoding nucleotides.

Info

Publication number
MX2021001901A
MX2021001901A MX2021001901A MX2021001901A MX2021001901A MX 2021001901 A MX2021001901 A MX 2021001901A MX 2021001901 A MX2021001901 A MX 2021001901A MX 2021001901 A MX2021001901 A MX 2021001901A MX 2021001901 A MX2021001901 A MX 2021001901A
Authority
MX
Mexico
Prior art keywords
factor
polynucleotides
viral particles
encoding nucleotides
compositions
Prior art date
Application number
MX2021001901A
Other languages
Spanish (es)
Inventor
Amit Nathwani
Jenny Mcintosh
Original Assignee
Ucl Business Ltd
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
Priority claimed from GBGB1813528.5A external-priority patent/GB201813528D0/en
Priority claimed from US16/105,583 external-priority patent/US10842885B2/en
Application filed by Ucl Business Ltd filed Critical Ucl Business Ltd
Publication of MX2021001901A publication Critical patent/MX2021001901A/en

Links

Classifications

    • 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
    • A61K38/46—Hydrolases (3)
    • 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
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—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
    • 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
    • C12N2830/007—Vector systems having a special element relevant for transcription cell cycle specific enhancer/promoter combination

Abstract

The present invention relates to polynucleotides comprising a Factor IX nucleotide sequence, wherein the Factor IX nucleotide sequence comprises a coding sequence that encodes a Factor IX protein or fragment thereof and wherein a portion of the coding sequence is not wild type. The present invention further relates to viral particles comprising a recombinant genome comprising the polynucleotide of the invention, compositions comprising the polynucleotides or viral particles, and methods and uses of the polynucleotides, viral particles or compositions.
MX2021001901A 2018-08-20 2019-08-20 Factor ix encoding nucleotides. MX2021001901A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1813528.5A GB201813528D0 (en) 2018-08-20 2018-08-20 Factor IX encoding nucleotides
US16/105,583 US10842885B2 (en) 2018-08-20 2018-08-20 Factor IX encoding nucleotides
PCT/GB2019/052339 WO2020039183A1 (en) 2018-08-20 2019-08-20 Factor ix encoding nucleotides

Publications (1)

Publication Number Publication Date
MX2021001901A true MX2021001901A (en) 2021-05-27

Family

ID=67810978

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021001901A MX2021001901A (en) 2018-08-20 2019-08-20 Factor ix encoding nucleotides.

Country Status (11)

Country Link
EP (1) EP3810770A1 (en)
JP (1) JP2021533799A (en)
KR (1) KR20210049129A (en)
CN (1) CN112739818A (en)
AU (1) AU2019323747A1 (en)
BR (1) BR112021002998A2 (en)
CA (1) CA3109579A1 (en)
IL (1) IL280973A (en)
MX (1) MX2021001901A (en)
SG (1) SG11202101560QA (en)
WO (1) WO2020039183A1 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
CN117693593A (en) * 2021-06-23 2024-03-12 英斯培瑞有限公司 Compositions and methods for treating hemophilia B
AU2022379633A1 (en) * 2021-10-27 2024-04-11 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE549037T1 (en) * 2004-09-22 2012-03-15 St Jude Childrens Res Hospital IMPROVED EXPRESSION OF FACTOR-IX IN GENE THERAPY VECTORS
ES2940323T3 (en) * 2008-09-15 2023-05-05 Uniqure Biopharma B V Factor IX polypeptide mutant, its uses and a method for its production
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
CA2865676C (en) * 2012-04-17 2020-03-10 Stefan Klostermann Method for the expression of polypeptides using modified nucleic acids
CN103667346B (en) * 2013-12-17 2016-01-20 扬州大学 A kind of pRBE-HCR-hAAT-hFIXml plasmid and structure thereof and application
US11008561B2 (en) * 2014-06-30 2021-05-18 Bioverativ Therapeutics Inc. Optimized factor IX gene
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
GB201508025D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
BR112017027956A2 (en) * 2015-06-23 2018-08-28 Childrens Hospital Philadelphia modified ix factor and compositions, methods and uses for gene transfer to cells, organs and tissues
CN116732066A (en) * 2016-10-14 2023-09-12 四川至善唯新生物科技有限公司 Preparation and application of high-activity blood coagulation factor IX mutant, recombinant protein and fusion protein

Also Published As

Publication number Publication date
IL280973A (en) 2021-04-29
AU2019323747A1 (en) 2021-03-04
EP3810770A1 (en) 2021-04-28
SG11202101560QA (en) 2021-03-30
WO2020039183A1 (en) 2020-02-27
KR20210049129A (en) 2021-05-04
CN112739818A (en) 2021-04-30
JP2021533799A (en) 2021-12-09
CA3109579A1 (en) 2020-02-27
BR112021002998A2 (en) 2021-05-11

Similar Documents

Publication Publication Date Title
MX2021001901A (en) Factor ix encoding nucleotides.
MX2017016105A (en) Adenovirus polynucleotides and polypeptides.
MY187559A (en) Modified hepatitis post-transcriptional regulatory elements
MX2017016610A (en) Methods of reducing odor.
PH12018501628A1 (en) Optimized factor viii genes
MX2020000495A (en) Encapsulated polynucleotides and methods of use.
SG11201810612TA (en) Novel genetically engineered vaccinia viruses
MX2018012265A (en) T cell receptors.
MX2021008146A (en) Encapsulated rna polynucleotides and methods of use.
BR112016018598A2 (en) adeno-associated virus vector
WO2014118360A3 (en) Carbohydrate degrading polypeptide and uses thereof
EA201300060A1 (en) POLYPEPTIDE, OWNED OR CONTRIBUTING TO THE ACTIVITY OF CARBON MATERIAL DEGRADATION AND ITS APPLICATION
SA518391787B1 (en) Composition for treatment of crigler-najjar syndrome
MX2018016114A (en) Novel heat-resistant fructose-6-phosphate-3-epimerase and method for producing allulose by using same.
EA201890351A1 (en) RECOMBINANT ORF-VIRAL VECTOR
MX2022011394A (en) Compositions and methods for inducing an immune response.
MX2019009551A (en) Lipolytic enzyme for use in baking.
WO2021084276A3 (en) Factor viii construct
EP3988653A3 (en) Udp-glycosyltransferases from solanum lycopersicum
EP3978614A3 (en) Single-vector gene construct comprising insulin and glucokinase genes
MX2022001028A (en) African swine fever vaccine.
MX2018003162A (en) Copy number variant leading to virus resistance.
MX2021009305A (en) Polynucleotides.
MX2015013448A (en) Polypeptides with lipase activity and polynucleotides encoding same.
MX2021014856A (en) Tgf-beta vaccine.