Patent SG-11201909453R-A (SG)
Title: Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family
Inventor: STEVIS PANAYIOTIS
IPC: C12Q1/32
Filed: 2018-03-06
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 18 October 2018 (18.10.2018) W I PO I PCT o mons °nolo VIII VIII 110111110 mil (10) International Publication Number WO 2018/190970 Al (51) International Patent Classification: C12Q 1/32 (2006.01) (21) International Application Number: PCT/US2018/021109 (22) International Filing Date: 06 March 2018 (06.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/484,141 11 April 2017 (11.04.2017) US (71) Applicant: REGENERON PHARMACEUTICALS, INC. [US/US]; 777 Old Saw Mill River Road, Tarrytown, New York 10591-6707 (US). (72) Inventors: STEVIS, Panayiotis; 777 Old Saw Mill Riv- er Road, Tarrytown, New York 10591-6707 (US). ESOPI, David; 777 Old Saw Mill River Road, Tarrytown, New York 10591-6707 (US). GROMADA, Jesper; 777 Old Saw Mill River Road, Tarrytown, New York 10591-6707 (US). HALLER, Jorge; 777 Old Saw Mill River Road, Tarrytown, New York 10591-6707 (US). LIU, Yashu; 777 Old Saw Mill River Road, Tarrytown, New York 10591-6707 (US). MURPHY, Andrew; 777 Old Saw Mill River Road, New York, New York 10591-6707 (US). OLSON, William; 777 Old Saw Mill River Road, Tarry- town, New York 10591-6707 (US). (74) Agent: LEGAARD, Paul K.; Stradley Ronon Stevens & Young, LLP, 30 Valley Stream Parkway, Malvern, Penn- sylvania 19355-1481 (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, 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. (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). Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(ii)) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Published: with international search report (Art. 21(3)) with sequence listing part of description (Rule 5.2(a)) 1-1 1-1 O N O 1-1 00 O N C (54) Title: ASSAYS FOR SCREENING ACTIVITY OF MODULATORS OF MEMBERS OF THE HYDROXYSTEROID (17-BE- TA) DEHYDROGENASE (HSD17B) FAMILY (57) : Screening methods as well as kits for identifying modulators of hydroxysteroid (17-beta) dehydrogenase (HSD17B) family member proteins, such as HSD17B13, are provided. The methods comprise screening molecules for their capacity to modulate the HSD17B family member protein, including inhibiting the HSD17B family member protein, as measured by substrate depletion, product concentration from the HSD17B family member protein substrate conversion or NADH concentration, levels of labeled sub- strate, luciferin light emission, or combinations thereof. Inhibitors of HSD17B family member proteins identified through the screening methods may be used to treat liver diseases, disorders, or conditions in which the HSD17B family member protein plays a role.