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1. (WO2017036889) METHOD FOR REDUCING IMMUNOGENICITY OF RNA
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Pub. No.: WO/2017/036889 International Application No.: PCT/EP2016/070012
Publication Date: 09.03.2017 International Filing Date: 24.08.2016
IPC:
A61K 48/00 (2006.01) ,C12N 15/10 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
10
Processes for the isolation, preparation or purification of DNA or RNA
Applicants:
BIONTECH RNA PHARMACEUTICALS GMBH [DE/DE]; An der Goldgrube 12 55131 Mainz, DE
Inventors:
KARIKÓ, Katalin; US
SAHIN, Ugur; DE
Agent:
SCHNAPPAUF, Georg; DE
Priority Data:
PCT/EP2015/06976028.08.2015EP
Title (EN) METHOD FOR REDUCING IMMUNOGENICITY OF RNA
(FR) PROCÉDÉ DE RÉDUCTION DE L'IMMUNOGÉNICITÉ D'ARN
Abstract:
(EN) The present invention relates to RNA therapy and, in particular, decreasing immunogenicity of RNA. Specifically, the present invention provides methods for decreasing immunogenicity of RNA, said methods comprising modifying the nucleotide sequence of the RNA by reducing the uridine (U) content, wherein said reduction of the U content comprises an elimination of U nucleosides from the nucleotide sequence of the RNA and/or a substitution of U nucleosides by nucleosides other than U in the nucleotide sequence of the RNA. Using RNA having decreased immunogenicity allows administration of RNA as a drug to a subject, e.g. in order to obtain expression of a pharmaceutically active peptide or protein, without eliciting an immune response which would interfere with therapeutic effectiveness of the RNA or induce adverse effects in the subject.
(FR) La présente invention concerne une thérapie par ARN et, en particulier, la diminution de l'immunogénicité d'un ARN. Spécifiquement, la présente invention concerne des procédés pour diminuer l'immunogénicité d'ARN, lesdits procédés comprenant la modification de la séquence nucléotidique de l'ARN, par diminution de la teneur en uridine (U), ladite diminution de la teneur en U comprenant une élimination de nucléosides U de la séquence nucléotidique de l'ARN et/ou une substitution de nucléosides U par des nucléosides autres que U dans la séquence nucléotidique de l'ARN. L'utilisation d'ARN ayant une immunogénicité réduite permet l'administration d'ARN en tant que médicament à un patient, par exemple afin d'obtenir l'expression d'un peptide ou une protéine pharmaceutiquement actif, sans induire une réponse immunitaire qui interfèrerait avec l'efficacité thérapeutique de l'ARN ou induirait des effets indésirables chez le patient.
Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)