SG11201808398PA - Cell line for recombinant protein and/or viral vector production - Google Patents

Cell line for recombinant protein and/or viral vector production

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Publication number
SG11201808398PA
SG11201808398PA SG11201808398PA SG11201808398PA SG11201808398PA SG 11201808398P A SG11201808398P A SG 11201808398PA SG 11201808398P A SG11201808398P A SG 11201808398PA SG 11201808398P A SG11201808398P A SG 11201808398PA SG 11201808398P A SG11201808398P A SG 11201808398PA
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SG
Singapore
Prior art keywords
international
rule
vectors
clone
cell line
Prior art date
Application number
SG11201808398PA
Inventor
Jingmin Zhou
Guang Qu
John Fraser Wright
Original Assignee
Spark Therapeutics Inc
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Publication date
Application filed by Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of SG11201808398PA publication Critical patent/SG11201808398PA/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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0684—Cells of the urinary tract or kidneys
    • C12N5/0686—Kidney cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C12N15/09—Recombinant DNA-technology
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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
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0012—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7)
    • C12N9/0026—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on CH-NH groups of donors (1.5)
    • C12N9/0028—Oxidoreductases (1.) acting on nitrogen containing compounds as donors (1.4, 1.5, 1.6, 1.7) acting on CH-NH groups of donors (1.5) with NAD or NADP as acceptor (1.5.1)
    • C12N9/003—Dihydrofolate reductase [DHFR] (1.5.1.3)
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    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/93—Ligases (6)
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    • C12Y—ENZYMES
    • C12Y105/00—Oxidoreductases acting on the CH-NH group of donors (1.5)
    • C12Y105/01—Oxidoreductases acting on the CH-NH group of donors (1.5) with NAD+ or NADP+ as acceptor (1.5.1)
    • C12Y105/01003—Dihydrofolate reductase (1.5.1.3)
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    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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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
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C12N2015/8518—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic expressing industrially exogenous proteins, e.g. for pharmaceutical use, human insulin, blood factors, immunoglobulins, pseudoparticles
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/70—Enzymes
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    • C12N2510/00—Genetically modified cells
    • C12N2510/02—Cells for production
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00011—Details
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07042—[Glutamate--ammonia-ligase] adenylyltransferase (2.7.7.42)
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    • C12Y—ENZYMES
    • C12Y603/00—Ligases forming carbon-nitrogen bonds (6.3)
    • C12Y603/01—Acid-ammonia (or amine)ligases (amide synthases)(6.3.1)
    • C12Y603/01002—Glutamate-ammonia ligase (6.3.1.2)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 111111110111101110101011111010111110111010111111111011111001111101111011111 International Bureau ... ..51 j (10) International Publication Number (43) International Publication Date .... ...r\";,' WO 2017/173043 Al 5 October 2017 (05.10.2017) IN I PO I P CT (51) International Patent Classification: (74) Agent: BEDGOOD, Robert M.; PILLSBURY WIN- G/2N 9/22 (2006.01) C07K 14/705 (2006.01) THROP SHAW PITTMAN LLP, P.O. BOX 10500, C12N 15/62 (2006.01) A61K 48/00 (2006.01) McLean, Virginia 22102 (US). C12N 15/85 (2006.01) (81) Designated States (unless otherwise indicated, for every (21) International Application Number: kind of national protection available): AE, AG, AL, AM, PCT/US2017/024951 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, (22) International Filing Date: DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, 30 March 2017 (30.03.2017) HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, (25) Filing Language: English KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (26) Publication Language: English NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, (30) Priority Data: RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, 62/315,480 30 March 2016 (30.03.2016) US TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (71) Applicant: SPARK THERAPEUTICS, INC. [US/US]; 3737 Market Street, Suite 1300, Philadelphia, (84) Designated States (unless otherwise indicated, for every Pennsylvania 19104 (US). kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, (72) Inventors: ZHOU, Jingmin; 203 Glendale Road, Walling- TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, ford, Pennsylvania 19086 (US). QU, Guang; 22 Beaver TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, Dam Drive, Sicklerville, New Jersey 08081 (US). DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, WRIGHT, John Fraser; 68 River Birch Circle, Princeton, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, New Jersey 08540 (US). [Continued on next page] = (54) Title: CELL LINE FOR RECOMBINANT PROTEIN AND/OR VIRAL VECTOR PRODUCTION (57) : Cells and cell lines are disclosed that are able to produce FIG. 2 therapeutic proteins, antibodies, vectors, and viral vectors such as lentiviral vectors and adeno-associated viral (AAV) vectors. The cells and/or cell lines can have mutations or deletions in either one or both of the endogenous di- Packaging cell line based hydrofolate reductase (DHFR-/-) or glutamine synthetase (GS-/-) genes such that DHFR and/or GS expression or function is substantially reduced or = = — production system eliminated. = • A novel parental cell lines (DHFR-/-; GS-/-) created. = = • Clones containing multi-thousand rAAV-genomes isolated. = — = • rAAV productivity is comparable to current RB productivity = • Cell clones are stable on vector production 3.50E+11 , = 3.00E+11 -r = _ , a 2.50E•11 i g 1.00E+11: 1-1 en i i 1.50.11 1.00E•11 co 7r © en 5.00E.= A l_ . 0.00E+00 -F- [lane clone clone clone clone clone clon clone clone clone HEg 11 IN Il © ei O WO 2017/173043 Al MIDEDIMOMOIDEIRMEMOMMEINVIMIUMEHOIMIE SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, of inventorship (Rule 4.17(iv)) GW, KM, ML, MR, NE, SN, TD, TG). Published: Declarations under Rule 4.17: — as to applicant's entitlement to apply for a patent (Rule 4.1700) — as to the applicant's entitlement to claim the earlier application (Rule 4.17(iii)) and the be granted priority of with international 21(3)) search report (Art.
SG11201808398PA 2016-03-30 2017-03-30 Cell line for recombinant protein and/or viral vector production SG11201808398PA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662315480P 2016-03-30 2016-03-30
PCT/US2017/024951 WO2017173043A1 (en) 2016-03-30 2017-03-30 Cell line for recombinant protein and/or viral vector production

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US (1) US20190078099A1 (en)
EP (1) EP3436576A4 (en)
JP (2) JP2019509748A (en)
KR (1) KR102479894B1 (en)
CN (1) CN109563496B (en)
AU (2) AU2017244133A1 (en)
BR (1) BR112018070250A2 (en)
CA (1) CA3019126A1 (en)
MX (1) MX2018011928A (en)
SG (1) SG11201808398PA (en)
WO (1) WO2017173043A1 (en)

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