Patent SG-11201907474U-A (SG)
Title: Compositions useful in treatment of spinal muscular atrophy
Inventor: WILSON JAMES
IPC: C12N15/864
Filed: 2018-02-27
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 07 September 2018 (07.09.2018) WIPO I PCT oimiolo VIII °nolo VIII ioo Num iflo X11 ois (10) International Publication Number WO 2018/160585 A2 (51) International Patent Classification: C12N 15/864 (2006.01) (21) International Application Number: PCT/US2018/019996 (22) International Filing Date: Published: — without international search report and to be republished upon receipt of that report (Rule 48.2(g)) — with sequence listing part of description (Rule 5.2(a)) (25) Filing Language: (26) Publication Language: (30) Priority Data: 27 February 2018 (27.02.2018) English English 62/464,756 28 February 2017 (28.02.2017) US 62/515,902 06 June 2017 (06.06.2017) US 62/618,437 17 January 2018 (17.01.2018) US (71) Applicant: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA [US/US]; 3160 Chesnut Street, Suite 200, Philadelphia, PA 19104 (US). (72) Inventors: WILSON, James, M.; 1831 Delancey Street, Philadelphia, PA 19103 (US). HINDERER, Christian; 212 Brown Street, Apt. 8A, Philadelphia, PA 19123 (US). KATZ, Nathan; 1111 Hope Street, Unit 3, Stamford, CT 06907 (US). WANG, Qiang; 228 West Winona Street, 1st Floor, Philadelphia, PA 19144 (US). (74) Agent: KODROFF, Cathy, A. et al.; Howson & Howson LLP, 350 Sentry Parkway, Building 620, Suite 210, Blue Bell, PA 19422 (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, N 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). GC O (54) Title: COMPOSITIONS USEFUL IN TREATMENT OF SPINAL MUSCULAR ATROPHY 00 (57) : A rAAV vector is described herein which has an AAVhu68 capsid and at least one expression cassette in the capsid. The at least one expression cassette comprises nucleic acid sequences encoding a functional SMN protein and expression control sequences that direct expression of the SMN sequences in a host cell. Also provided are compositions containing this rAAVhu68.SMN vector and O N methods of using same for spinal muscular atrophy in a patient. O