Patent SG-11201901107R-A (SG)
Title: Methods and compositions relating to improved human red blood cell survival in genetically modified immunodeficient non-human animals
Inventor: SHULTZ LEONARD D
IPC: C12N15/63
Filed: 2017-08-11
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 111011110111010101111101111101001111111101111101010110111110111 OEN Organization International Bureau (10) International Publication Number (43) International Publication Date ......0\" WO 2018/031920 Al 15 February 2018 (15.02.2018) WIP0 I PCT (51) International Patent Classification: (74) Agent: STAPLE, Ph. D., Julie K. et al.; Dinsmore & Shohl A01K 67/02 (2006.01) A61K 49/00 (2006.01) LLP, 900 Wilshire Drive, Suite 300, Troy, MI 48084 (US). AO1K 67/027 (2006.01) Cl 2N 15/63 (2006.01) (81) Designated States (unless otherwise indicated, for every (21) International Application Number: kind of national protection available): AE, AG, AL, AM, PCT/US2017/046566 AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, (22) International Filing Date: CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, 11 August 2017 (11.08.2017) 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, (25) Filing Language: English KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, (26) Publication Language: English MG, MK, MN, WV, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, (30) Priority Data: SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, 62/373,671 11 August 2016 (11.08.2016) US TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (71) Applicant: THE JACKSON LABORATORY [US/US]; (84) Designated States (unless otherwise indicated, for every 610 Main Street, Bar Harbor, ME 04609 (US). kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, (72) Inventors: SHULTZ, Leonard D.; 80 Indian Point Rd., UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, Bar Harbor, ME 04609 (US). WILES, Michael V.; 9 Beech Hill Rd., Mount Desert, ME 04660 (US). 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, = (54) Title: METHODS AND COMPOSITIONS GENETICALLY MODIFIED IMMUNODEFICIENT NON-HUMAN ANIMALS = = = = loo = 80 = t71 = 60 — z _ 0 = 0 40 = CO = CC 20 = 0 % RELATING TO IMPROVED NSG vs NSG MD1 HUMAN RED BLOOD CELL SURVIVAL IN -.- NSG --0-- NSG MD1 0 10 20 30 Hours 1-1 FIG. I © N t:T 1-1 f .r1 (57) : A genetically modified immunodeficient non-human animal whose genome includes a genetic modification that renders 0 - the non-human animal deficient in macrophages and/or macrophage anti-human red blood cell activity so as to prolong the survival GC of human red blood cells when administered into said non-human animal is provided according to aspects of the present invention. 1-1 © Methods of assaying effects of putative therapeutic agents in such a genetically modified immunodeficient non-human animal are N provided by the present invention. O [Continued on next page] WO 2018/031920 Al MIDEDIMOMMIDIREI0111111210101011MOVOIS TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). 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 amendments (Rule 48.2(h)) of — with sequence listing part of description (Rule 5.2(a))