Patent SG-11201808812R-A (SG)
Title: Novel aav8 mutant capsids and compositions containing same
Inventor: WILSON JAMES
IPC: C12N15/864
Filed: 2017-04-13
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 11111111011110111010101111101011111011101111110111111111110011111111101111011111 International Bureau .. .... ..Yejd ..... ...„1 (10) International Publication Number (43) International Publication Date WO 2017/180854 Al 19 October 2017 (19.10.2017) WIPO I PCT (51) International Patent Classification: (74) Agents: SCHALLER, Colleen, M. et al.; Howson & C12N 15/86 (2006.01) C07K 7/06 (2006.01) Howson LLP, 350 Sentry Parkway, Building 620, Suite C12N 15/864 (2006.01) C07K 14/005 (2006.01) 210, Blue Bell, PA 19422 (US). (21) International Application Number: (81) Designated States (unless otherwise indicated, for every PCT/US2017/027392 kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, (22) International Filing Date: BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, 13 April 2017 (13.04.2017) DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, (25) Filing Language: English HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, (26) Publication Language: English MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (30) Priority Data: NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, 62/323,389 15 April 2016 (15.04.2016) US 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, (71) Applicant: THE TRUSTEES OF THE UNIVERSITY ZA, ZM, ZW. OF PENNSYLVANIA [US/US]; 3160 Chestnut Street, Suite 200, Philadelphia, PA 19104 (US). (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, (72) Inventors: WILSON, James, M.; 1831 Delancey Street, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, Philadelphia, PA 19103 (US). WANG, Qiang; 228 West TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, Winona Street, 1st Floor, Philadelphia, PA 19144 (US). 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, = [Continued on next page] = = (54) Title: NOVEL — — = _ = = = = = = AAV8 MUTANT CAPSIDS AND COMPOSITIONS CONTAINING SAME (57) : Provided herein are AAV8 mutant capsids and rAAV comprising the same. In one embodiment, vec - 5'-ITR tors employing the AAV8 mutant capsid show increased NheI transduction in a selected tissue as compared to AAV8. PolyA site m. i 0/P ----- ..-..--'' Zs-Green I PGK Promoter = Amp-R ir NheI = CMV promoter . — = N s .........____ 4, HindIII _ — 10 \" --- partial Rep = Aaarl 1-1 SpeI 3-ITR . 71' ir) GC © FIGURE lA cc ,-1 IN ,-1 © ei O WO 2017/180854 Al MIDEDIMOMMION010130MIMMIEHRUDIS SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, before the expiration of the time limit for amending the GW, KM, ML, MR, NE, SN, TD, TG). Published: claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) — with international search report (Art. 21(3)) — with sequence listing part of description (Rule 5.2(a))