Patent SG-11201804994Q-A (SG)
Title: Adeno-associated viral vectors for treating mucolipidosis type ii
Inventor: YEW NELSON
IPC: A01K67/027
Filed: 2016-12-14
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 1111111101111011101010111110101111101110101101101110110111001111101111011111 International Bureau 0.. .... .. (10) International Publication Number ...... ... (43) International Publication Date WO 2017/106313 Al 22 June 2017(22.06.2017) WIPO I PCT (51) International Patent Classification: AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, C12N 15/86 (2006.01) C12N 9/12 (2006.01) BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, A01K 67/027 (2006.01) A61K 48/00 (2006.01) 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, (21) International Application Number: KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, PCT/US2016/066611 MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (22) International Filing Date: NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, 14 December 2016 (14.12.2016) 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, (25) Filing Language: English ZA, ZM, ZW. (26) Publication Language: English (84) Designated States (unless otherwise indicated, for every (30) Priority Data: kind of regional protection available): ARIPO (BW, GH, 62/267,502 15 December 2015 (15.12.2015) 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) Applicants: GENZYME CORPORATION [US/US]; TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, 500 Kendall Street, Cambridge, MA 02142 (US). SAM- DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, SUNG LIFE PUBLIC WELFARE FOUNDATION LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, [KR/KR]; 48, Itaewon-ro 55-gil, Yongsan-gu, Seoul (KR). SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, (72) Inventors: YEW, Nelson; c/o Sanofi, 55 Corporate Drive, GW, KM, ML, MR, NE, SN, TD, TG). Bridgewater, NJ 08807 (US). JIN, Dong-Kyu; c/o Sam- Declarations under Rule 4.17: — sung Life Public Welfare Foundation, 48, Itaewon-ro 55- — of inventorship (Rule 4.17(N)) gil, Yongsan-gu, Seoul (KR). — Published: Agents: DONAHUE, Brian, A. et al.; Morrison & Foer- (74) _ ster LLP, 755 Page Mill Road, Palo Alto, CA 94304-1018 — with international search report (Art. 21(3)) = (U S) . — with sequence listing part of description (Rule 5.2(a)) = Designated States (unless otherwise indicated, for every = (81) kind of national protection available): AE, AG, AL, AM, = = = = = = = = = = = = 1-1 M 11 M 0 1-1 (54) Title: ADENO-ASSOCIATED VIRAL VECTORS FOR TREATING MUCOLIPIDOSIS TYPE II IN (57) : Provided herein are recombinant adeno-associated virus (rAAV) vectors comprising nucleic acid encoding N-acetyl- 1-1 c:::: ) glucosamine-1 -phosphate transferase, alpha and beta subunits (GNPTAB) and at least one AAV inverted terminal repeat (ITR). In ei some embodiments, the rAAV vectors may be included in a rAAV particle, which may be contained in pharmaceutical compositions O and kits. These vectors, particles, compositions, and kits may find use, inter alia, in methods and uses related to treating mucol - ipidosis type II (ML II) or mucolipidosis type III (ML III) in a mammal, or related to increasing body size, bone mineral content, and/or bone mineral density in a mammal with mucolipidosis type II (ML II) or mucolipidosis type III (ML III).