Patent SG-11201407529W-A (SG)
Title: High-transduction-efficiency raav vectors, compositions, and methods of use
Inventor: SRIVASTAVA ARUN
IPC: C12N15/864
Filed: 2013-05-15
Abstract: (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 21 November 2013 (21.11.2013) WIPOIPCT (10) International Publication Number WO 2013/173512 A3 (51) International Patent Classification: C07K14/05 (2006.01) A61K38/16 (2006.01) C12N15/35 (2006.01) A61K 48/00 (2006.01) CI2N15/864 (2006.01) (21) International Application Number: (22) International Filing Date: (25) Filing Language: (26) Publication Language: PCT/US2013/041234 15 May 2013 (15.05.2013) English (30) Priority Data: 61/647,318 13/840,224 15 May 2012 (15.05.2012) 15 March 2013 (15.03.2013) English US US (71) Applicant: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC. [US/US]; 223 Grinter Hall, Gainesville, Florida 32611 (US). (72) Inventors: SRIVASTAVA, Arun; 3823 SW 92nd Drive, Gainesville, Florida 32608 (US). ASLANIDI, George Vladimirovich; 2411 NW 69th Terrace, Gainesville, Flor­ ida 32626 (US). VAN VLIET, Kim M.; 8124 SW 57th Place, Gainesville, Florida 32608 (US). AGBAND- JE-McKENNA, Mavis; 10202 SW 17th Place, Gaines­ ville, Florida 32607 (US). (74) Agents: MOORE, Mark D. et al.; Haynes and Boone, LLP, 2323 Victory Avenue, Suite 700, Dallas, Texas 75219 (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, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IS, JP, KE, KG, KM, KN, KP, KR, KZ, LA, LC, LK, LR, LS, LT, 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, 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, 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, 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, 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 of amendments (Rule 48.2(h)) (88) Date of publication of the international search report: 10 April 2014 CJ i-H m i> i-H o CJ (54) Title: HIGH-TRANSDUCTION-EFFICIENCY RAAV VECTORS, COMPOSITIONS, AND METHODS OF USE (57) Abstract: The present invention provides AAV capsid proteins comprising modification of one or a combination of the surface- exposed lysine, serine, threonine and/or tyrosine residues in the VP3 region. Also provided are rAAV virions comprising the AAV capsid proteins of the present invention, as well as nucleic acid molecules and rAAV vectors encoding the AAV capsid proteins of the present invention. Advantageously, the rAAV vectors and virions of the present invention have improved efficiency in transduc - tion of variety a of cells, tissues and organs of interest, when compared to wild-type rAAV vectors and virions.