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1. WO2021092485 - COMPOSITIONS AND METHODS FOR TREATING SICKLE CELL DISEASE

Publication Number WO/2021/092485
Publication Date 14.05.2021
International Application No. PCT/US2020/059535
International Filing Date 06.11.2020
IPC
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
C07H 21/02 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
02with ribosyl as saccharide radical
C12N 5/10 2006.01
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
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
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
C12N 15/63 2006.01
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
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
C12Q 1/68 2018.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
1Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
68involving nucleic acids
Applicants
  • SYROS PHARMACEUTICALS, INC. [US]/[US]
Inventors
  • MCCARTY, Catherine M.
Agents
  • MCCARTY, Catherine, M.
Priority Data
62/931,69206.11.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS FOR TREATING SICKLE CELL DISEASE
(FR) COMPOSITIONS ET MÉTHODES DE TRAITEMENT DE LA DRÉPANOCYTOSE
Abstract
(EN)
The present invention features compositions and methods useful in inhibiting the expression of NFIX within a cell and thereby treating patients suffering from a hemoglobinopathy such as sickle cell disease or β-thalassemia. The present invention features, inter alia, methods of treating a hemoglobinopathy in a patient in need thereof. The methods can be carried out by administering to the patient an effective amount of a pharmaceutically acceptable composition comprising a genetically modified cell that comprises a nucleic acid construct that inhibits the expression of NFIX within the cell.
(FR)
La présente invention concerne des compositions et des procédés utiles pour inhiber l'expression de NFIX dans une cellule et traiter ainsi des patients atteints d'une hémoglobinopathie telle que la drépanocytose ou la β-thalassémie. La présente invention concerne, inter alia, des procédés de traitement d'une hémoglobinopathie chez un patient en ayant besoin. Les procédés peuvent être exécutés par administration au patient d'une quantité efficace d'une composition pharmaceutiquement acceptable comprenant une cellule génétiquement modifiée qui comporte une constructoin d'acide nucléique qui inhibe l'expression de NFIX à l'intérieur de la cellule.
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