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SG11201901941YA - High purity rna compositions and methods for preparation thereof - Google Patents

High purity rna compositions and methods for preparation thereof

Info

Publication number
SG11201901941YA
SG11201901941YA SG11201901941YA SG11201901941YA SG11201901941YA SG 11201901941Y A SG11201901941Y A SG 11201901941YA SG 11201901941Y A SG11201901941Y A SG 11201901941YA SG 11201901941Y A SG11201901941Y A SG 11201901941YA SG 11201901941Y A SG11201901941Y A SG 11201901941YA
Authority
SG
Singapore
Prior art keywords
ppp
international
street
rna
rna compositions
Prior art date
Application number
SG11201901941YA
Inventor
Stephen Hoge
William Issa
Edward J Miracco
Jennifer Nelson
Amy Rabideau
Gabor Butora
Original Assignee
Modernatx Inc
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.)
Filing date
Publication date
Application filed by Modernatx Inc filed Critical Modernatx Inc
Publication of SG11201901941YA publication Critical patent/SG11201901941YA/en

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04Antibacterial agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P33/00Antiparasitic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475Growth factors; Growth regulators
    • C07K14/505Erythropoietin [EPO]
    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00Preparation of compounds containing saccharide radicals
    • C12P19/26Preparation of nitrogen-containing carbohydrates
    • C12P19/28N-glycosides
    • C12P19/30Nucleotides
    • C12P19/34Polynucleotides, e.g. nucleic acids, oligoribonucleotides
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6803General methods of protein analysis not limited to specific proteins or families of proteins
    • G01N33/6848Methods of protein analysis involving mass spectrometry
    • G01N33/6851Methods of protein analysis involving laser desorption ionisation mass spectrometry

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Spectroscopy & Molecular Physics (AREA)
  • Virology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 111111111111111111111111111111111111111111111111111111111111111 111111111 01111 Organization International Bureau (43) International Publication Date . . .., . .. (10) International Publication Number WO 2018/053209 Al 22 March 2018 (22.03.2018) WIP0 I PCT (51) International Patent Classification: MA 02451 (US). BUTORA, Gabor; 200 Technology Cl 2N 15/10 (2006.01) Square, Cambridge, MA 02139 (US). (21) International Application Number: (74) Agent: LOCKHART, Helen C.; Wolf, Greenfield & PCT/US2017/051674 Sacks, P.C., 600 Atlantic Avenue, Boston, MA 02210-2206 (22) International Filing Date: (US). 14 September 2017 (14.09.2017) (81) Designated States (unless otherwise indicated, for every (25) Filing Language: English kind AO, of national protection available): AE, AG, AL, AM, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, (26) Publication Language: English CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, (30) Priority Data: DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, 62/394,711 14 September 2016 (14.09.2016) US HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, (71) Applicant: MODERNATX, INC. [US/US]; 320 Bent MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, Street, Cambridge, MA 02141 (US). 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, (72) Inventors: HOGE, Stephen; 320 Bent Street, Cambridge, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. MA 02141 (US). ISSA, William; 95 Kimball Road, Ded- ham, MA 02026 (US). MIRACCO, Edward J.; 6 Frost (84) Designated States (unless otherwise indicated, for every Street, Arlington, MA 02474 (US). NELSON, Jennifer; kind of regional protection available): ARIPO (BW, GH, — 465 Washington Street, #2, Brookline, MA 02246 (US). GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, RABIDEAU, Amy, E.; 121 Pond Street, Unit 1, Waltham, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, = = (54) Title: HIGH PURITY RNA COMPOSITIONS AND METHODS FOR PREPARATION THEREOF = = Figure 40 = = = = DNA plasmid = NTPs = = Linearized dsDNA template 2+ = Mg — = = = 5 'PPP - ,-- -\",, .........vappp 5' = 5 '13PP 5 'PPP -A ino NN... 3' Full-length product Run-on transcripts Reverse Abortive/ truncated transcripts 1 complement ,.._ impurities , = 1€ 5'PPP ;€€€€€€€ 5 'P P :.1 5'PPP EM 113 A ioo N N... 3' wituunsww-ppp 5' samonsitutu-ppp 5' Ennwansmeam-ppp 5' meWmammtata-ppp 5' ireamfamaros-PPP 5' CD . IMIH liJilli © ei PP P 5' Kw:mamma-pp p 5' M 0 i n (57) : The invention relates to improved RNA compositions for use in therapeutic applications. The RNA compositions are ---- particularly suited for use in human therapeutic application (e.g., in RNA therapeutics). The RNA compositions are made by inproved *1: processes, in particular, improved in vitro-transcription (IVT) processes. The invention also relates to methods for producing and 1-1 © purifying RNA (e.g, therapeutic RNAs), as well as methods for using the RNA compositions and therapeutic applications thereof. ei O [Continued on next page] WO 2018/053209 Al MIDEDIMOMOIDEIREEMONIMEINEMECIONVOIS 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)) — with sequence listing part of description (Rule 5.2(a))
SG11201901941YA 2016-09-14 2017-09-14 High purity rna compositions and methods for preparation thereof SG11201901941YA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662394711P 2016-09-14 2016-09-14
PCT/US2017/051674 WO2018053209A1 (en) 2016-09-14 2017-09-14 High purity rna compositions and methods for preparation thereof

Publications (1)

Publication Number Publication Date
SG11201901941YA true SG11201901941YA (en) 2019-04-29

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201901941YA SG11201901941YA (en) 2016-09-14 2017-09-14 High purity rna compositions and methods for preparation thereof

Country Status (11)

Country Link
US (4) US11202793B2 (en)
EP (2) EP4166666A1 (en)
JP (2) JP6983455B2 (en)
CN (2) CN109937253B (en)
AU (2) AU2017326423B2 (en)
CA (1) CA3036831A1 (en)
ES (1) ES2928475T3 (en)
MA (1) MA46240A (en)
MX (1) MX2019002904A (en)
SG (1) SG11201901941YA (en)
WO (1) WO2018053209A1 (en)

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