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1. WO2020113112 - SYSTEMS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES

Publication Number WO/2020/113112
Publication Date 04.06.2020
International Application No. PCT/US2019/063766
International Filing Date 27.11.2019
IPC
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
A61K 31/7125 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61K 31/712 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
C12N 5/10 2006.01
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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
5Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
A61P 7/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
7Drugs for disorders of the blood or the extracellular fluid
CPC
C12N 15/113
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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 2310/20
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2310Structure or type of the nucleic acid
10Type of nucleic acid
20involving clustered regularly interspaced short palindromic repeats [CRISPRs]
C12N 2310/315
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2310Structure or type of the nucleic acid
30Chemical structure
31of the backbone
315Phosphorothioates
C12N 2310/346
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2310Structure or type of the nucleic acid
30Chemical structure
34Spatial arrangement of the modifications
346having a combination of backbone and sugar modifications
C12N 2320/31
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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
2320Applications; Uses
30Special therapeutic applications
31Combination therapy
Applicants
  • EDITAS MEDICINE, INC. [US]/[US]
Inventors
  • AUPEPIN DE LAMOTHE-DREUZY, Edouard
  • CHANG, KaiHsin
  • HEATH, Jack
Agents
  • MORRIS, Patrick
  • PROCHNOW, Courtney
Priority Data
62/773,08929.11.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) SYSTEMS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
(FR) SYSTÈMES ET MÉTHODES POUR LE TRAITEMENT D’HÉMOGLOBINOPATHIES
Abstract
(EN)
Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci, portions of the erythroid specific enhancer of the BCL11A gene, or a combination thereof, in cells and increasing expression of fetal hemoglobin.
(FR)
L’invention concerne des systèmes d'édition de génome, des ARN guides et des méthodes à médiation par CRISPR pour modifier des parties des loci des gènes HBG1 et HBG2, des parties de la séquence activatrice spécifique de l’érythroïde du gène BCL11A, ou une combinaison de celles-ci, dans des cellules et pour augmenter l'expression de l'hémoglobine fœtale.
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