Patent SG-11201809388S-A (SG)
Title: Relative potency assay for viral vector encoding isomerohydrolases
Inventor: COUTO LINDA
IPC: C12Q1/00
Filed: 2017-04-28
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 02 November 2017 (02.11.2017) WI P0 I PC T IiiimmomiolollmolommolommoilommuniovoimIE (10) International Publication Number WO 2017/190081 Al (51) International Patent Classification: A61K 31/07 (2006.01) Cl 2N 15/86 (2006.01) C12N 9/18 (2006.01) C12Q 1/00 (2006.01) (21) International Application Number: PCT/US2017/030254 (22) International Filing Date: 28 April 2017 (28.04.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/328,916 28 April 2016 (28.04.2016) US (71) Applicant: SPARK THERAPEUTICS, INC. [US/US]; 3737 Market Street, Suite 1300, Philadelphia, PA 19104 (US). (72) Inventor: COUTO, Linda; 7814 Oak Creek Drive, Pleasanton, CA 94588 (US). (74) Agent: BEDGOOD, Robert, M.; Pillsbury Winthrop Shaw Pittman LLP, P.O. Box 10500, McLean, VA 22102 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): 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, 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. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, 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). (54) Title: RELATIVE POTENCY ASSAY FOR VIRAL VECTOR ENCODING ISOMERHYDROLASES Figure 7 Use of assay to demonstrat. different Assay tiesigin 3 HEK293/tRAT cells transduced with two different lOts AAV,ii+Ei6 a Five MOIS (3S1(r, 1%10 4 , 3X10, 11410, 3x10' vector genomesicell) • Cell lysates analyzed for RPE65 activity w 4 Parameter Logistic Model used to Compare two lots of steam Conclusion: The hotor and ProlwOUSci (enmities) are. both 'cos, Indicattribbhat there amino differences between the curves. Thus, two lots d : re simila, pas...1m.y 1-1 GC N (57) : Methods for assaying function and/or activity and/or potency of isomerohydrolase proteins are provided. [Continued on next page] O WO 2017/190081 Al MIDEDIMOMMIMOIDEMOMOHMONEHHINEVOIMIE Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(H)) 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))