SG11201807378SA - Trans-replicating rna - Google Patents

Trans-replicating rna

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Publication number
SG11201807378SA
SG11201807378SA SG11201807378SA SG11201807378SA SG11201807378SA SG 11201807378S A SG11201807378S A SG 11201807378SA SG 11201807378S A SG11201807378S A SG 11201807378SA SG 11201807378S A SG11201807378S A SG 11201807378SA SG 11201807378S A SG11201807378S A SG 11201807378SA
Authority
SG
Singapore
Prior art keywords
rna
international
present
pct
mainz
Prior art date
Application number
SG11201807378SA
Inventor
Tim Beissert
Ugur Sahin
Mario Perkovic
Original Assignee
Biontech Rna Pharmaceuticals Gmbh
Tron Translationale Onkologie An Der Univ Der Johannes Gutenberg Univ Mainz Gemeinnuetzige Gmbh
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Publication date
Application filed by Biontech Rna Pharmaceuticals Gmbh, Tron Translationale Onkologie An Der Univ Der Johannes Gutenberg Univ Mainz Gemeinnuetzige Gmbh filed Critical Biontech Rna Pharmaceuticals Gmbh
Publication of SG11201807378SA publication Critical patent/SG11201807378SA/en

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/16—Antivirals for RNA viruses for influenza or rhinoviruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C12N15/1131—Non-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 against viruses
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/09—Recombinant DNA-technology
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/127—RNA-directed RNA polymerase (2.7.7.48), i.e. RNA replicase
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07048—RNA-directed RNA polymerase (2.7.7.48), i.e. RNA replicase
    • C—CHEMISTRY; METALLURGY
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    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/36011—Togaviridae
    • C12N2770/36111—Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/36011—Togaviridae
    • C12N2770/36111—Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36141—Use of virus, viral particle or viral elements as a vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/36011—Togaviridae
    • C12N2770/36111—Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki
    • C12N2770/36141—Use of virus, viral particle or viral elements as a vector
    • C12N2770/36143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Biochemistry (AREA)
  • Virology (AREA)
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  • Physics & Mathematics (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Communicable Diseases (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pulmonology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (10) International Publication Number (43) International Publication Date WO 2017/162461 Al 28 September 2017 (28.09.2017) WIPO I PCT (51) International Patent Classification: C12N 15/86 (2006.01) C12N 5/10 (2006.01) C12N 15/40 (2006.01) A61K 48/00 (2006.01) (21) International Application Number: PCT/EP2017/055813 (22) International Filing Date: 13 March 2017 (13.03.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: PCT/EP2016/056160 21 March 2016 (21.03.2016) EP (71) Applicants: BIONTECH RNA PHARMACEUTICALS GMBH [DE/DE]; An der Goldgrube 12, 55131 Mainz (DE). TRON - TRANSLATIONALE ONKOLOGIE AN DER UNIVERSITATSMEDIZIN DER JOHANNES GUTENBERG-UNIVERSITAT MAINZ GEMEIN- NUTZIGE GMBH [DE/DE]; Freiligrathstr. 12, 55131 Mainz (DE). (72) Inventors: BEISSERT, Tim; Edith-Stein-Weg 4, 64521 Gross-Gerau (DE). SAHIN, Ugur; Philipp-von-Za- bern-Platz 1, 55116 Mainz (DE). PERKOVIC, Mario; Falkenweg 1, 60437 Frankfurt (DE). (74) Agent: SCHNAPPAUF, Georg; ZSP Patentanwalte PartG mbB, Hansastrasse 32, 80686 Miinchen (DE). (81) Designated States (unless otherwise indicated, for every kind of national protection available): 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, DJ, 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, KH, KN, KP, KR, KW, 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. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (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), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: — with international search report (Art. 21(3)) = (54) Title: TRANS-REPLICATING RNA Fig. 1 A SFV genome SGP (incl C\"E3) CSE4 MINV., aped & glyeopostelns -7300-1 -3700nt -11400nt cis-replicon L Gene of interest CSE1,2 SGP iCSE4 --poIyA -770.0nt transreplicon replicase encoding mRNA CSE1S2 SGP te , CSE4 polyA -1500nt (57) : The present invention generally relates to systems and methods suitable for high-level protein production. While one or more elements of the present invention are derived from an alphavirus, the present invention does not require propagation of virus particles. In particular, a system comprising two separate RNA molecules is foreseen, each comprising a nucleotide sequence derived from an alphavirus: one RNA molecule comprises a RNA construct for expressing alphavirus replicase, and one RNA molecule comprises a RNA re pi icon that can be replicated by the replicase in trans. The RNA construct for expressing alphavirus replicase comprises a 5'-cap. It was surprisingly found that the 5'-cap is suitable for efficiently driving expression of a transgene from the repl - icon in trans. The system of the present invention enables expression of a protein of interest in a cell or organism, but is not associ - ated with undesired virus-particle formation. Therefore, the present invention is suitable for efficiently and safely producing a pro - tein of interest, e.g. a therapeutic protein or an antigenic protein, such as a vaccine, in a target organism. Respective methods of pro - tein production in vitro and in vivo as well as medical uses are provided herein. The present invention also provides DNA encoding the RNA molecules of the invention, and cells comprising the RNA molecules of the invention. 1111111111111101110111111111110101111101110111111110101111111111011111111111110111111 W O 20 17 / 16 246 1 Al
SG11201807378SA 2016-03-21 2017-03-13 Trans-replicating rna SG11201807378SA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
PCT/EP2016/056160 WO2017162265A1 (en) 2016-03-21 2016-03-21 Trans-replicating rna
PCT/EP2017/055813 WO2017162461A1 (en) 2016-03-21 2017-03-13 Trans-replicating rna

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Publication Number Publication Date
SG11201807378SA true SG11201807378SA (en) 2018-10-30

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US (1) US20200299724A1 (en)
EP (2) EP3964584A1 (en)
JP (2) JP7105193B2 (en)
KR (1) KR102441725B1 (en)
CN (2) CN115386596A (en)
AU (1) AU2017236240B2 (en)
BR (1) BR112018068391A2 (en)
CA (1) CA3017285A1 (en)
CY (1) CY1124689T1 (en)
DK (1) DK3433368T3 (en)
ES (1) ES2896927T3 (en)
HR (1) HRP20211715T1 (en)
HU (1) HUE058889T2 (en)
IL (1) IL261327B1 (en)
LT (1) LT3433368T (en)
MA (1) MA44479B1 (en)
MD (1) MD3433368T2 (en)
MX (1) MX2018011384A (en)
PL (1) PL3433368T3 (en)
PT (1) PT3433368T (en)
RS (1) RS62543B1 (en)
RU (1) RU2752580C2 (en)
SG (1) SG11201807378SA (en)
SI (1) SI3433368T1 (en)
WO (2) WO2017162265A1 (en)
ZA (2) ZA201805520B (en)

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