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1. (WO2018226887) ENHANCING AGENTS FOR IMPROVED CELL TRANSFECTION AND/OR rAAV VECTOR PRODUCTION
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Pub. No.: WO/2018/226887 International Application No.: PCT/US2018/036344
Publication Date: 13.12.2018 International Filing Date: 06.06.2018
IPC:
C12N 15/09 (2006.01) ,C12N 15/63 (2006.01) ,C12N 15/86 (2006.01) ,A61K 47/60 (2017.01)
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
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
63
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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
63
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79
Vectors or expression systems specially adapted for eukaryotic hosts
85
for animal cells
86
Viral vectors
[IPC code unknown for A61K 47/60]
Applicants:
SPARK THERAPEUTICS, INC. [US/US]; 3737 Market Street, Suite 1300 Philadelphia, Pennsylvania 19104, US
Inventors:
QU, Guang; US
LU, Lin; US
JOSUE-ALMQVIST, Jesusa; US
WRIGHT, John Fraser; US
Agent:
BEDGOOD, Robert M.; US
Priority Data:
62/516,43207.06.2017US
62/531,62612.07.2017US
Title (EN) ENHANCING AGENTS FOR IMPROVED CELL TRANSFECTION AND/OR rAAV VECTOR PRODUCTION
(FR) AGENTS D’ACTIVATION DESTINÉS À LA TRANSFECTION DE CELLULES ET/OU LA PRODUCTION DE VECTEUR RAAV AMÉLIORÉES
Abstract:
(EN) Provided are compositions and methods of transducing/transfecting cells with a molecule, such as a nucleic acid (e.g., plasmid), at high efficiency. High efficiency transduced/transfected cells can, when transduced with a nucleic acid that encodes a protein or comprises a sequence that is transcribed into a transcript of interest, produce high amounts of protein and/or transcript. High efficiency transduced/transfected cells can, when transduced with plasmids comprising (i) nucleic acids encoding AAV packaging proteins and/or nucleic acids encoding helper proteins; and (ii) a transgene that encodes a protein or is transcribed into a transcript of interest; produce high amounts of recombinant rAAV vector.
(FR) La présente invention concerne des compositions et des procédés de transduction/transfection de cellules avec une molécule, telle qu’un acide nucléique (par exemple, un plasmide), à un rendement élevé. Les cellules transduites/transfectées à rendement élevé peuvent, lorsqu’elles sont transduites avec un acide nucléique qui code pour une protéine ou qui comprend une séquence qui est transcrite dans un produit de transcription d’intérêt, produire des quantités élevées de protéine et/ou de produit de transcription. Les cellules transduites/transfectées à rendement élevé peuvent, lorsqu’elles sont transduites avec des plasmides comprenant (i) des acides nucléiques codant pour les protéines d’encapsulation AAV et/ou des acides nucléiques codant pour des protéines auxiliaires ; et (ii) un transgène qui code pour une protéine ou est transcrit dans un produit de transcription d’intérêt ; produire des quantités élevées de vecteur rAAV recombinant.
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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, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, 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, 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)