Patent SG-11201900366Q-A (SG)
Title: Scalable high recovery methods for producing high yield recombinant adeno-associated viral (raav) vector and recombinant adeno-associated viral (raav) vectors produced thereby
Inventor: QU GUANG
IPC: C12N15/861
Filed: 2017-07-21
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 111111110111101110101011111011111011111111010111111111111011101111101111001111 Organization - - - ' International Bureau (10) International Publication Number (43) International Publication Date .....0\" WO 2018/017956 A3 25 January 2018 (25.01.2018) WI P0 I P C T (51) International Patent Classification: Pennsylvania 19010 (US). WANG, Yuhuan; 37 Buck- C12N 15/861 (2006.01) C07K 2/00 (2006.01) ingham Place, Cherry Hill, New Jersey 08003-2665 (US). C12N 7/00 (2006.01) Cl 2N 15/64 (2006.01) ZHANG, Haibo; 30 President Avenue, Rutledge, Penn- A61K 48/00 (2006.01) sylvania 19070 (US). DUNCAN, Laura; 50 South Valley (21) International Application Number: Road, Apt. G-06, Paoli, Pennsylvania 19301 (US). PCT/US2017/043291 (74) Agent: BEDGOOD, Robert M.; PILLSBURY WIN- THROP SHAW PITTMAN LLP, P.O. BOX 10500, (22) International Filing Date: McLean, Virginia 22102 (US). 21 July 2017 (21.07.2017) (25) Filing Language: English (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (26) Publication Language: English AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, (30) Priority Data: CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, 62/365,312 21 July 2016 (21.07.2016) US DZ, EC, EE, EG, ES, FL GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, (71) Applicant: SPARK THERAPEUTICS, INC. [US/US]; KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, 3737 Market Street, Suite 1300, Philadelphia, Pennsylvania MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, 19104 (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: QU, Guang; 22 Beaver Dam Drive, Sick-TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. lerville, New Jersey 08081 (US). WRIGHT, John Fras- er; 68 River Birch Circle, Princeton, New Jersey 08540 (84) Designated States (unless otherwise indicated, for every = = (US). OH, Younghoon; 766 Robinhood Road, Bryn Mawr, kind of regional protection available): ARIPO (BW, GH, = = (54) Title: SCALABLE HIGH RECOVERY METHODS FOR PRODUCING HIGH YIELD RECOMBINANT ADENO-ASSOCIAT- ED VIRAL (RAAV) VECTOR AND RECOMBINANT ADENO-ASSOCIATED VIRAL (RAAV) VECTORS PRODUCED THERE- = BY 1 17 95 6 A3 1 11 111 1111111111 111111 11111 1 1111111 111111 111111 1 111111111 11 11 A 5.00E+13 4.50E+13 4.00E+13 3.50E+13 2 ---- 3.00E+13 1 3 2.50E+13 '8 2.00E+13 1.50E+13 1.00E+13 5.00E+12 aopEioo \" , \..._....... ' \ ./ r' ‘ ‘..\ \., \ K ,,,,, titer (vg/mL) \ r---------------------- diameter ..- % -------- 35 • ‘`-% ' 30 2 • '\" r.. , E 20 8 1 2 1 n . ii 0 -- -- - -- - -- - - - - (nm) . fit 3000 sec-1 1.38E+13 ii t 3000 sec-1 : 35.77 11E10000 sec-Ii 4.81E+13 . IN 10000 sec-1. 36.47 1 Figure 1 ---.... Il © (57) : Provided are methods for producing recombinant adeno-associated virus (rAAV) vector particles at high recovery or ei high titer. Also provided are methods that concentrate rAAV vectors to a high concentration, for example, up to 5E+13 (5x10 13 ) vector C genomes per milliliter (Vg/ml) with little if any rAAV aggregates. [Continued on next page] WO 2018/017956 A3 IMEDIMOM0101011MERIONIMMIIHRIMODEfin GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, 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, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Declarations under Rule 4.17: — as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(U)) — as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) — of inventorship (Rule 4.17(iv)) Published: — with international search report (Art. 21(3)) — before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) (88) Date of publication of the international search report: 08 March 2018 (08.03.2018)