Patent SG-11201907606X-A (SG)
Title: Humanized model of kidney and liver disorders
Inventor: DEVALARAJA-NARASHIMHA KISHOR
IPC: C07K14/47
Filed: 2018-02-26
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 30 August 2018 (30.08.2018) WIPO I PCT onion °nolo HE loonliolommo oimIE (10) International Publication Number WO 2018/157027 Al (51) International Patent Classification: A61K 49/00 (2006.01) CO7K 14/47 (2006.01) A01K 67/027 (2006.01) G01N 33/50 (2006.01) (21) International Application Number: PCT/US2018/019651 (22) International Filing Date: 26 February 2018 (26.02.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/464,262 27 February 2017 (27.02.2017) US 62/529,916 07 July 2017 (07.07.2017) US 62/583,780 09 November 2017 (09.11.2017) US (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 N N O N kr) 00 O N C (71) Applicant: REGENERON PHARMACEUTICALS, INC. [US/US]; 777 Old Saw Mill River Road, Tarrytown, NY 10591-6707 (US). (72) Inventors: DEVALARAJA-NARASHIMHA, Kishor; c/ o Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY 10591-6707 (US). MOR- TON, Lori; c/o Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY 10591-6707 (US). LUO, Yifan; c/o Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY 10591-6707 (US). HUANG, Cong; c/o Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY 10591-6707 (US). MEAGHER, Karolina; c/o Regeneron Pharmaceu- ticals, Inc., 777 Old Saw Mill River Road, Tarrytown, NY 10591-6707 (US). CASANOVA, Sarah; c/o Regeneron Pharmaceuticals, Inc., 777 Old Saw Mill River Road, Tar- rytown, NY 10591-6707 (US). (74) Agent: GROLZ, Edward, W.; Scully, Scott, Murphy & Presser, 400 Garden City Plaza, Suite 300, Garden City, NY 11530 (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. (54) Title: HUMANIZED MODEL OF KIDNEY AND LIVER DISORDERS (57) : Provided herein, inter alia, are non-human animals comprising nucleic acid sequences encoding a C3 protein that com- prises a human sequence as well as transgenic non-human animals comprising a C3 gene that is human in whole or in part as well as methods for using the same to screen for candidate therapeutic molecules to treat complement-related nephropathies. Also provided herein are methods for improving kidney function in an individual diagnosed with or thought to have a complement-related nephropathy.