Patent SG-11201808398P-A (SG)
Title: Cell line for recombinant protein and/or viral vector production
Inventor: ZHOU JINGMIN
IPC: C12N15/62
Filed: 2017-03-30
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.