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1. WO2020118115 - COMPOSITIONS AND METHODS FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY

Publication Number WO/2020/118115
Publication Date 11.06.2020
International Application No. PCT/US2019/064786
International Filing Date 05.12.2019
IPC
C12N 9/10 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
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
10Transferases (2.)
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
C12N 15/86 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
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
CPC
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
A61P 9/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
9Drugs for disorders of the cardiovascular system
C12N 9/1018
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
9Enzymes; Proenzymes; Compositions thereof
10Transferases (2.)
1003transferring one-carbon groups (2.1)
1018Carboxy- and carbamoyl transferases (2.1.3)
C12Y 201/03003
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
YENZYMES
201Transferases transferring one-carbon groups (2.1)
03Carboxy- and carbamoyltransferases (2.1.3)
03003Ornithine carbamoyltransferase (2.1.3.3)
Applicants
  • ARCTURUS THERAPEUTICS, INC. [US]/[US]
Inventors
  • PEREZ-GARCIA, Carlos, G.
  • TACHIKAWA, Kiyoshi
  • MATSUDA, Daiki
  • CHIVUKULA, Padmanabh
Agents
  • SHERIN, Mridula, P.
  • ALLISON, William, F.
  • AMENDT, Kevin, C.
  • ADAMS, Lisa
  • AZRIN, Linda, B.
Priority Data
62/776,30206.12.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY
(FR) COMPOSITIONS ET MÉTHODES POUR TRAITER UNE DÉFICIENCE EN ORNITHINE TRANSCARBAMYLASE
Abstract
(EN)
The present disclosure provides a modified human OTC protein having improved properties for the treatment of OTC deficiency in a patient. Preferably, the protein of the disclosure is produced from a codon optimized mRNA suitable for administration to a patient suffering from OTC deficiency wherein upon administration of the mRNA to the patient, the protein of the disclosure is expressed in the patient in therapeutically effective amounts to treat OTC deficiency. The present disclosure also provides codon optimized mRNA sequences encoding wild type human OTC comprising a 5' UTR derived from a gene expressed by Arabidopsis thaliana for use in treating OTC deficiency in a patient.
(FR)
La présente invention concerne une protéine OTC humaine modifiée ayant des propriétés améliorées pour le traitement d'une déficience en OTC chez un patient. De préférence, la protéine de l'invention est produite à partir d'un ARNm optimisé en termes de codons adaptée pour l'administration à un patient souffrant d'une déficience en OTC, l'administration de l'ARNm au patient conduisant à l'expression de la protéine de l'invention chez le patient dans des quantités thérapeutiquement efficaces pour traiter la déficience en OTC. La présente invention concerne également des séquences d'ARNm optimisées en termes de codons, codant pour l'OTC humaine de type sauvage comprenant une 5'UTR dérivée d'un gène exprimé par Arabidopsis thaliana pour une utilisation dans le traitement d'une déficience en OTC chez un patient.
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