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1. WO2020118239 - MODIFIED PROTEINS AND ASSOCIATED METHODS OF TREATMENT

Publication Number WO/2020/118239
Publication Date 11.06.2020
International Application No. PCT/US2019/065046
International Filing Date 06.12.2019
IPC
C07K 14/435 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
C12Q 1/37 2006.01
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
34involving hydrolase
37involving peptidase or proteinase
Applicants
  • ARCTURUS THERAPEUTICS, INC. [US]/[US]
Inventors
  • PEREZ-GARCIA, Carlos, G.
  • CHIVUKULA, Padmanabh
  • TACHIKAWA, Kiyoshi
Agents
  • SHERIN, Mridula, P.
  • ALLISON, Wiiliam, F.
  • AZRIN, Linda, B.
  • AMENDT, Kevin, C
  • ADAMS, Lisa
Priority Data
62/776,32206.12.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) MODIFIED PROTEINS AND ASSOCIATED METHODS OF TREATMENT
(FR) PROTÉINES MODIFIÉES ET MÉTHODES DE TRAITEMENT ASSOCIÉES
Abstract
(EN)
The present disclosure provides a modified human protein having improved in vivo stability. The modified human protein has been altered from a wild-type human protein at either at least one ubitquitination site, at the signal peptide portion, or both. The protein of the disclosure can be produced from a codon optimized mRNA suitable for administration to a patient suffering from deficiency of the wild-type protein, wherein upon administration of the mRNA to the patient, the modified protein of the disclosure is expressed in the patient in therapeutically effective amounts.
(FR)
La présente invention concerne une protéine humaine modifiée ayant une stabilité in vivo stability améliorée. La protéine humaine modifiée a été modifiée à partir d'une protéine humaine de type sauvage au niveau d'au moins un site d'ubiquitination et/ou au niveau de la partie peptide signal. La protéine de l'invention peut être produite à partir d'un ARNm optimisé en termes de codons approprié pour l'administration à un patient souffrant d'une déficience de la protéine de type sauvage, l'administration de l'ARNm au patient conduisant à l'expression chez le patient de la protéine modifiée de l'invention dans des quantités thérapeutiquement efficaces.
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