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1. WO2020065303 - EDITING OF HAEMOGLOBIN GENES

Publication Number WO/2020/065303
Publication Date 02.04.2020
International Application No. PCT/GB2019/052696
International Filing Date 25.09.2019
IPC
A61K 35/28 2015.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
A61K 48/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/805 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
795Porphyrin- or corrin-ring-containing peptides
805Haemoglobins; Myoglobins
C12N 15/113 2010.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
CPC
A61K 35/28
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
A61K 38/42
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
41Porphyrin- or corrin-ring-containing peptides
42Haemoglobins; Myoglobins
A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C07K 14/805
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
795Porphyrin- or corrin-ring-containing peptides
805Haemoglobins; Myoglobins
C12N 15/113
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
C12N 15/907
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
90Stable introduction of foreign DNA into chromosome
902using homologous recombination
907in mammalian cells
Applicants
  • OXFORD UNIVERSITY INNOVATION LIMITED [GB]/[GB]
Inventors
  • DAVIES, James
  • HIGGS, Douglas
  • BADAT, Mohsin
Agents
  • DEHNS
Priority Data
1815670.326.09.2018GB
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) EDITING OF HAEMOGLOBIN GENES
(FR) ÉDITION DE GÈNES D'HÉMOGLOBINE
Abstract
(EN)
The present invention relates to a process for producing a modified nucleic acid, wherein the nucleic acid comprises a mutant haemoglobin B (HBB) gene encoding a mutant Hb-β polypeptide. The process comprises using a base editor, preferably with a gRNA, to edit the mutant HBBgene to change a first (mutant) codon in that gene into a second, non-wild-type codon, wherein the Hb-β polypeptide encoded by that edited HBBgene has a non-wild-type, yet phenotypically-viable, amino acid sequence. The invention also provides a population of isolated haematopoietic stem cells,the stem cells comprising edited HBBgenes.
(FR)
La présente invention concerne un procédé de production d'un acide nucléique modifié, l'acide nucléique comprenant un gène d'hémoglobine B mutant (HBB) codant pour un polypeptide Hb-β mutant. Le procédé comprend l'utilisation d'un éditeur de bases, comportant de préférence un ARNg, pour éditer le gène HBB mutant afin de transformer un premier codon (mutant) de ce gène en un second codon de type non sauvage, le polypeptide Hb-β codé par ce gène HBB édité comportant une séquence d'acides aminés de type non sauvage, mais phénotypiquement viable. L'invention concerne également une population de cellules souches hématopoïétiques isolées, les cellules souches comprenant des gènes HBB édités.
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