Patent SG-11201809029R-A (SG)
Title: Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy
Inventor: RODINO-KLAPAC LOUISE
IPC: C12N15/85
Filed: 2017-04-14
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 1111111101111011101010111110101111101110111111011011110111111101111101111011111 International Bureau .. .... ..Yejd .... ...,,,' (10) International Publication Number (43) International Publication Date WO 2017/180976 Al 19 October 2017(19.10.2017) WIPO I PCT (51) International Patent Classification: (81) Designated States (unless otherwise indicated, for every A61K 48/00 (2006.01) C12N 15/12 (2006.01) kind of national protection available): AE, AG, AL, AM, A61P 21/00 (2006.01) C12N 15/85 (2006.01) AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, C07K 14/47 (2006.01) BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FL GB, GD, GE, GH, GM, GT, (21) International Application Number: HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, PCT/US2017/027583 KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, (22) International Filing Date: MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, 14 April 2017 (14.04.2017) 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, (25) Filing Language: English TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, (26) Publication Language: English ZA, ZM, ZW. (30) Priority Data: (84) Designated States (unless otherwise indicated, for every 62/323,333 15 April 2016 (15.04.2016) US kind of regional protection available): ARIPO (BW, GH, 62/433,548 13 December 2016 (13.12.2016) US GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, (71) Applicant: RESEARCH INSTITUTE AT NATION- TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, WIDE CHILDREN'S HOSPITAL [US/US]; 700 Chil- DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, dren's Drive, Room W172, Columbus, OH 43205 (US). LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CL CM, GA, GN, GQ, (72) Inventors: RODINO-KLAPAC, Louise; 4912 Bixby Ridge Drive E, Groveport, OH 43125 (US). MENDELL, GW, KM, ML, MR, NE, SN, TD, TG). Jerry, R.; 8176 Copperfield Drive, Columbus, OH 43235 Published: (US). — with international search report (Art. 21(3)) — (74) Agents: SINTICH, Sharon M. et al.; Marshall, Gerstein with sequence listing of description (Rule 5.2(a)) part & Borun LLP, 233 S. Wacker Drive, 6300 Willis Tower, Chicago, IL 60606-6357 (US). — with information concerning incorporation by reference of missing parts and/or elements (Rule 20.6) Il IN C:r © GC 11 (54) Title: ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF B-SARCOGLYCAN AND MICRORNA-29 AND THE 11 TREATMENT OF MUSCULAR DYSTROPHY 0 N (57) : Described herein are recombinant AAV vectors comprising a polynucleotide sequence comprising [3-sarcoglycan and O methods of using the recombinant vectors to reduce or prevent fibrosis in a mammalian subject suffering from a muscular dystrophy. Also described herein are combination therapies comprising administering AAV vector(s) expressing [3-sarcoglycan and miR-29c to a mammalian subject suffering from a muscular dystrophy.