Patent SG-11201907714U-A (SG)
Title: Adeno-associated virus (aav) clade f vector and uses therefor
Inventor: WILSON JAMES
IPC: C12N7/00
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 ill 1111u°111110111sOHolo momioollm 11111E11mo oimIE (10) International Publication Number WO 2018/160582 Al (51) International Patent Classification: C12N 7/00 (2006.01) A61K 48/00 (2006.01) A61K 35/76 (2015.01) (21) International Application Number: PCT/US2018/019992 (22) International Filing Date: 27 February 2018 (27.02.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/464,748 28 February 2017 (28.02.2017) US 62/591,002 27 November 2017 (27.11.2017) US 62/614,002 05 January 2018 (05.01.2018) US 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). 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)) with sequence listing part of description (Rule 5.2(a)) (71) Applicant: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA [US/US]; 3160 Chestnut Street, Suite 200, Philadaelphia, PA 19104 (US). (72) Inventors: WILSON, James, M.; 1831 Delancey Street, Philadelphia, PA 19103 (US). WANG, Qiang; 228 West Winona Street, 1st Floor, Philadelphia, PA 19144 (US). GILES, April; 844 N. 29th Street, Apt 121, Philadelphia, PA 19130 (US). TURNER, Kevin; 124 Beechwood Road, Newtown Square, PA 19073 (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, cc O 00 O O (54) Title: ADENO-ASSOCIATED VIRUS (AAV) CLADE F VECTOR AND USES THEREFOR (57) : A recombinant adeno-associated virus (rAAV) vector comprising an AAVhu68 capsid produced in a production system comprising a nucleotide sequence of SEQ ID NO: 1, or a sequence at least 75% identical thereto which encodes SEQ ID NO:2. The AAVhu68 capsid comprises subpopulations of highly deamidated asparagine residues in asparagine - glycine pairs in the amino acid sequence of SEQ ID NO: 2. Also provided are compositions containing the rAAV and uses thereof. Additionally, rAAV having an engineered AAV capsid comprising at least one subpopulation of vpl or vp2 proteins having a Val at amino acid position 157 with reference to the AAVhu68 vpl numbering are provided.