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HRP20220607T1 - Terminalno modificirana rna - Google Patents

Terminalno modificirana rna Download PDF

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Publication number
HRP20220607T1
HRP20220607T1 HRP20220607TT HRP20220607T HRP20220607T1 HR P20220607 T1 HRP20220607 T1 HR P20220607T1 HR P20220607T T HRP20220607T T HR P20220607TT HR P20220607 T HRP20220607 T HR P20220607T HR P20220607 T1 HRP20220607 T1 HR P20220607T1
Authority
HR
Croatia
Prior art keywords
formulation
nanoparticles according
lipid nanoparticles
mrna
microrna
Prior art date
Application number
HRP20220607TT
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English (en)
Inventor
Tirtha Chakraborty
Stephane Bancel
Stephen G. HOGE
Atanu Roy
Antonin De Fougerolles
Noubar B. Afeyan
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Modernatx, Inc.
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=49447811&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HRP20220607(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Modernatx, Inc. filed Critical Modernatx, Inc.
Publication of HRP20220607T1 publication Critical patent/HRP20220607T1/hr

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67General methods for enhancing the expression
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection

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  • General Engineering & Computer Science (AREA)
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  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)

Claims (16)

1. Formulacija lipidnih nanočestica mRNA za ciljanu ekspresiju polipeptida od interesa u specifičnim tipovima stanica, navedena mRNA koja sadrži (a) neprevedeno područje 5’ (b) regija povezanih nukleozida koja kodira polipeptid od interesa; (c) neprevedeno područje 3' koje sadrži najmanje jedno vezno mjesto mikroRNA za mikroRNA izraženu u specifičnom tipu stanice tako da se ekspresija mRNA može smanjiti, pri čemu je mRNA usmjerena na razgradnju ili smanjenu translaciju u prisutnosti mikroRNA; i (d) repno područje 3' povezanih nukleozida, pri čemu je uracil ili uridin u mRNA zamijenjen do 100% modificiranim uracilom ili modificiranim uridinom, odnosno.
2. Formulacija lipidnih nanočestica prema zahtjevu 1, naznačena time što bilo koja od regija (a) - (d) u zahtjevu 1 sadrži najmanje jedan analog pseudouridina, pri čemu je izborno analog pseudouridina 1-metilpseudouridin u kojem slučaju mRNA izborno dodatno sadrži modificirani nukleozid 5-metilcitidin.
3. Formulacija lipidnih nanočestica prema zahtjevu 1, naznačena time što je barem jedno područje mRNA optimizirano kodonom, izborno pri čemu je područje povezanih nukleozida koji kodiraju navedeni polipeptid od interesa optimiziran za kodon.
4. Formulacija lipidnih nanočestica prema zahtjevu 1, naznačena time što je 5' UTR nativni 5'UTR kodiranog polipeptida.
5. Formulacija lipidnih nanočestica prema zahtjevu 1, naznačena time što 5'UTR sadrži sekvencu inicijacije translacije odabranu iz skupine koja se sastoji od Kozakove sekvence i unutarnjeg ulaznog mjesta ribosoma (IRES).
6. Formulacija lipidnih nanočestica prema zahtjevu 1, naznačena time što je 5'UTR strukturirani UTR.
7. Formulacija lipidnih nanočestica prema zahtjevu 1, koja sadrži najmanje jednu strukturu kape 5', naznačena time što je izborno najmanje jedna struktura kape 5' odabrana iz skupine koja se sastoji od Cap0, Cap1, ARCA, inozin, N1-metil-gvanozin, 2'fluor-gvanozin, 7-deaza-gvanozin, 8-okso-gvanozin, 2-amino-gvanozin, LNA- gvanozin, 2-azido-gvanozin, Cap2, Cap4, i CAP-003 - CAP-225.
8. Formulacija lipidnih nanočestica prema zahtjevu 1, naznačena time što je najmanje jedno vezno mjesto mikroRNA za specifičnu imunološku stanicu mikroRNA, pri čemu je izborno specifična imunološka stanica mikroRNA odabrana iz skupine koja se sastoji od miR-122, miR-142-3p, miR-142-5p, miR-146a i miR-146b.
9. Formulacija lipidnih nanočestica prema zahtjevu 1, naznačena time što repno područje 3' povezanih nukleozida nadalje sadrži nukleozid završnog lanca, pri čemu je izborno nukleozid završnog lanca odabran iz skupine koja se sastoji od 3'-deoksiadenozina (kordicepin), 3'-deoksiuridina, 3'-deoksicitozina, 3'-deoksigvanozina, 3'-deoksitimina, 2',3'-dideoksinukleozida, 2',3'-dideoksiadenozina, 2',3'-dideoksiuridina, 2',3'-dideoksicitozina, 2',3'-dideoksiguanozina, 2',3'-dideoksitimina, 2'-deoksinukleozida i -O-metilnukleozida.
10. Formulacija lipidnih nanočestica prema zahtjevu 1, naznačena time što repno područje 3' sadrži sekvencu petlje stabljike.
11. Formulacija lipidnih nanočestica prema zahtjevu 1, naznačena time što 5'UTR sadrži najmanje jednu sekvencu elementa za pojačavanje translacije (TEE).
12. Formulacija lipidnih nanočestica prema bilo kojem od prethodnih zahtjeva, naznačena time što je najmanje jedno vezno mjesto mikroRNA za miR-142-3p.
13. Formulacija lipidnih nanočestica prema bilo kojem od prethodnih zahtjeva, naznačena time što mRNA sadrži najmanje dva vezna mjesta mikroRNA.
14. Formulacija lipidnih nanočestica prema zahtjevu 13, naznačena time što su vezna mjesta mikroRNA ista.
15. Formulacija lipidnih nanočestica prema zahtjevu 13, naznačena time što su vezna mjesta mikroRNA različita.
16. Formulacija lipidnih nanočestica mRNA kako je definirana u bilo kojem od zahtjeva 1-15 za uporabu u terapijskoj metodi gdje je ekspresija mRNA usmjerena na specifične tipove stanica.
HRP20220607TT 2012-11-26 2013-10-02 Terminalno modificirana rna HRP20220607T1 (hr)

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US201261729933P 2012-11-26 2012-11-26
US201261737224P 2012-12-14 2012-12-14
US201361758921P 2013-01-31 2013-01-31
US201361775509P 2013-03-09 2013-03-09
US201361781139P 2013-03-14 2013-03-14
US201361829372P 2013-05-31 2013-05-31
US201361829359P 2013-05-31 2013-05-31
US201361839903P 2013-06-27 2013-06-27
US201361842709P 2013-07-03 2013-07-03
US201361857436P 2013-07-23 2013-07-23
EP13779667.8A EP2922554B1 (en) 2012-11-26 2013-10-02 Terminally modified rna
PCT/US2013/062943 WO2014081507A1 (en) 2012-11-26 2013-10-02 Terminally modified rna

Publications (1)

Publication Number Publication Date
HRP20220607T1 true HRP20220607T1 (hr) 2022-06-24

Family

ID=49447811

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HRP20220607TT HRP20220607T1 (hr) 2012-11-26 2013-10-02 Terminalno modificirana rna

Country Status (16)

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US (5) US9597380B2 (hr)
EP (2) EP2922554B1 (hr)
JP (5) JP6144355B2 (hr)
AU (3) AU2017202228B2 (hr)
CA (1) CA2892529C (hr)
CY (1) CY1125236T1 (hr)
DK (1) DK2922554T3 (hr)
ES (1) ES2921623T3 (hr)
HR (1) HRP20220607T1 (hr)
LT (1) LT2922554T (hr)
PL (1) PL2922554T3 (hr)
PT (1) PT2922554T (hr)
RS (1) RS63237B1 (hr)
SI (1) SI2922554T1 (hr)
SM (1) SMT202200337T1 (hr)
WO (1) WO2014081507A1 (hr)

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