CA3109579A1 - Nucleotides codant pour le facteur ix - Google Patents

Nucleotides codant pour le facteur ix Download PDF

Info

Publication number
CA3109579A1
CA3109579A1 CA3109579A CA3109579A CA3109579A1 CA 3109579 A1 CA3109579 A1 CA 3109579A1 CA 3109579 A CA3109579 A CA 3109579A CA 3109579 A CA3109579 A CA 3109579A CA 3109579 A1 CA3109579 A1 CA 3109579A1
Authority
CA
Canada
Prior art keywords
codons
encode
factor
sequence
polynucleotide
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3109579A
Other languages
English (en)
Inventor
Amit Nathwani
Jenny MCINTOSH
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
UCL Business Ltd
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 CA3109579A1 publication Critical patent/CA3109579A1/fr
Pending legal-status Critical Current

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
    • 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
    • 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)
    • 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
    • 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
    • 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)
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

La présente invention concerne des polynucléotides comprenant une séquence nucléotidique du facteur IX, la séquence nucléotidique du facteur IX comprenant une séquence codante qui code une protéine du facteur IX ou un fragment de celle-ci et une partie de la séquence codante n'étant pas de type sauvage. La présente invention concerne en outre des particules virales comprenant un génome recombinant comprenant le polynucléotide de l'invention, des compositions comprenant les polynucléotides ou les particules virales, et des procédés et des utilisations des polynucléotides, des particules virales ou des compositions.
CA3109579A 2018-08-20 2019-08-20 Nucleotides codant pour le facteur ix Pending CA3109579A1 (fr)

Applications Claiming Priority (5)

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
US16/105,583 2018-08-20
GB1813528.5 2018-08-20
PCT/GB2019/052339 WO2020039183A1 (fr) 2018-08-20 2019-08-20 Nucléotides codant pour le facteur ix

Publications (1)

Publication Number Publication Date
CA3109579A1 true CA3109579A1 (fr) 2020-02-27

Family

ID=67810978

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3109579A Pending CA3109579A1 (fr) 2018-08-20 2019-08-20 Nucleotides codant pour le facteur ix

Country Status (11)

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

Families Citing this family (4)

* 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
EP4361278A1 (fr) * 2021-06-23 2024-05-01 Inspirar Limited Composition et procédé de traitement de l'hémophilie b
US20230149563A1 (en) * 2021-10-27 2023-05-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
WO2024170976A1 (fr) * 2023-02-18 2024-08-22 Intas Pharmaceuticals Ltd. Polynucléotide à codon optimisé codant pour le facteur ix humain

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1804839B1 (fr) * 2004-09-22 2012-03-14 St. Jude Children's Research Hospital Expression améliorée du facteur ix dans les vecteurs de thérapie génique
EP4219547A3 (fr) * 2008-09-15 2023-10-18 uniQure biopharma B.V. Mutant polypeptidique du facteur ix, ses utilisations et son procédé de production
WO2011122950A1 (fr) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Vecteurs aav duplex monomériques
JP6224077B2 (ja) * 2012-04-17 2017-11-01 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 改変された核酸を用いてポリペプチドを発現させるための方法
CN103667346B (zh) * 2013-12-17 2016-01-20 扬州大学 一种pRBE-HCR-hAAT-hFIXml质粒及其构建和应用
EP3160478A4 (fr) * 2014-06-30 2018-05-16 Bioverativ Therapeutics Inc. Gène du facteur ix optimisé
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
WO2016210170A1 (fr) * 2015-06-23 2016-12-29 The Children's Hospital Of Philadelphia Facteur ix modifié, et compositions, méthodes et utilisations pour un transfert de gènes dans des cellules, des organes et des tissus
CN116355883A (zh) * 2016-10-14 2023-06-30 四川至善唯新生物科技有限公司 一种高活性凝血因子ix突变体、重组蛋白与融合蛋白的制备与应用

Also Published As

Publication number Publication date
KR20210049129A (ko) 2021-05-04
EP3810770A1 (fr) 2021-04-28
SG11202101560QA (en) 2021-03-30
WO2020039183A1 (fr) 2020-02-27
BR112021002998A2 (pt) 2021-05-11
AU2019323747A1 (en) 2021-03-04
CN112739818A (zh) 2021-04-30
MX2021001901A (es) 2021-05-27
IL280973A (en) 2021-04-29
JP2021533799A (ja) 2021-12-09

Similar Documents

Publication Publication Date Title
US11491213B2 (en) Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues
US20220331409A1 (en) Factor ix gene therapy
JP6473416B2 (ja) 第ｖｉｉｉ因子配列
CA3109579A1 (fr) Nucleotides codant pour le facteur ix
US20230226224A1 (en) Polynucleotide
US11517631B2 (en) Factor IX encoding nucleotides
US12209262B2 (en) Factor IX encoding nucleotides
US20210395714A1 (en) Modified factor ix polypeptides
GB2576508A (en) Factor IX encoding nucleotides
US20240076691A1 (en) Codon-optimized nucleic acid encoding the fix protein
Mcintosh et al. Factor VIII sequences
HK1252351B (en) Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues