Patent SG-11201808354P-A (SG)
Title: Column-based fully scalable raav manufacturing process
Inventor: QU GUANG
IPC: A61K38/46
Filed: 2017-03-31
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 11111111011110111010101111101011111011101011111001011111111001111101111011111 International Bureau ... ..... ..)d (10) International Publication Number (43) International Publication Date ..... .....::„I WO 2017/173283 Al 5 October 2017 (05.10.2017) IN I PO I P CT (51) International Patent Classification: (US). WRIGHT, John Fraser; 68 River Birch Circle, A61K 38/46 (2006.01) Cl 2N 15/86 (2006.01) Princeton, New Jersey 08540 (US). (21) International Application Number: (74) Agent: BEDGOOD, Robert M.; Pillsbury Winthrop Shaw PCT/US2017/025396 Pittman LLP, P.O. Box 10500, McLean, Virginia 22102 (US). (22) International Filing Date: 31 March 2017 (31.03.2017) (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (25) Filing Language: English AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, (26) Publication Language: English BZ, DO, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, (30) Priority Data: HN, 62/316,252 31 March 2016 (31.03.2016) US KP, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, (71) Applicant: SPARK THERAPEUTICS, INC. [US/US]; MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, 3737 Market Street, Suite 1300, Philadelphia, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, Pennsylvania 19104 (US). RU, TH, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, (72) Inventors: QU, Guang; 22 Beaver Dam Drive, Sick- ZA, ZM, ZW. lerville, New Jersey 08081 (US). OH, Younghoon; 107 Lowrys Lane, Bryn Mawr, Pennsylvania 19010 (US). LU, (84) Designated States (unless otherwise indicated, for every Lin; 439 Robin Hill Road, Wayne, Pennsylvania 19087 kind GM, of regional protection available): ARIPO (BW, GH, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, [Continued on next page] (54) Title: COLUMN-BASED FULLY SCALABLE RAAV MANUFACTURING PROCESS (57) : In accordance with the invention, provided FIG. 3 herein are methods for purifying recombinant adeno-associ- ated (rAAV) vector particles. All column purification process 1 2 3 4 98 62 vpl vp2 vp3 49 1. AVB 2. AVB > CIEX > AIEX Il 3. AVB AIEX > 4. CIEX > UF/DF > SEC > en ot AIEX (50-60 M ei 1 7 50-60% vector recovery IN 1-1 IN 1-1 0 ei 0 WO 2017/173283 Al MIDEDIMOMOIDEIRMEMOMMEINIMMOOMEHOIS TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, — as to the applicant's entitlement to claim the priority of TJ, 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, — the earlier application (Rule 4.17(iii)) of inventorship (Rule 4.17(iv)) SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, Published: GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). — with international search report Declarations under Rule 4.17: (Art. 21(3)) — as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(ii))