Patent SG-11201811244S-A (SG)
Title: Dual overlapping adeno-associated viral vector system for expressing abc4a
Inventor: MACLAREN ROBERT
IPC: C12N15/86
Filed: 2017-06-14
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 21 December 2017 (21.12.2017) WIP0 I PCT Iiiimmomoiollmolommiolominiloolionv oimIE (10) International Publication Number WO 2017/216560 Al (51) International Patent Classification: Cl 2N 15/86 (2006.01) (21) International Application Number: PCT/GB2017/051741 (22) International Filing Date: 14 June 2017 (14.06.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 1610448.1 15 June 2016 (15.06.2016) GB 1707261.2 05 May 2017 (05.05.2017) GB (71) Applicant: OXFORD UNIVERSITY INNOVATION LIMITED [GB/GB]; Buxton Court, 3 West Way, Botley Oxford OX2 OJB (GB). (72) Inventors: MACLAREN, Robert; Nuffield Laboratory of Ophthalmology, Levels 5 & 6, West Wing, John Rad- cliffe Hospital, Headley Way, Headington Oxford OX3 9DU (GB). MCCLEMENTS, Michelle; Nuffield Labora- tory of Ophthalmology, Levels 5 & 6, West Wing, John Radcliffe Hospital, Headley Way, Headington Oxford OX3 9DU (GB). (74) Agent: D YOUNG & CO LLP; 120 Holborn, London EC1N 2DY (GB). (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, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, (54) Title: DUAL OVERLAPPING ADENO-ASSOCIATED VIRAL VECTOR SYSTEM FOR EXPRESSING ABC4A ITR Promoter ABCA4 CDS ITR Upstream transgene Region of over ap Downstream transgene ITR ABCA4 CDS WPRE pA ITR Region of overlap enables recombination of the upstream and downstream transgenes to form a single, larger transgene with the complete ABCA4 CDS TR Promoter ABCA4 CDS WPRE pA 1-1 O 1-1 N _ N O 1-1 N C FIG. 10 (57) : The present invention provides an adeno-associated viral (AAV) vector system for expressing a human ABCA4 protein in a target cell, the AAV vector system comprising a first AAV vector comprising a first nucleic acid sequence and a second AAV vector comprising a second nucleic acid sequence; wherein the first nucleic acid sequence comprises a 5' end portion of an ABCA4 coding sequence (CDS) and the second nucleic acid sequence comprises a 3' end portion of an ABCA4 CDS, and the 5' end portion and the 3' end portion together encompass the entire ABCA4 CDS; wherein the first nucleic acid sequence comprises a sequence of contiguous nucleotides corresponding to nucleotides 105 to 3597 of SEQ ID NO: 1; wherein the second nucleic acid sequence comprises a sequence of contiguous nucleotides corresponding to nucleotides 3806 to 6926 of SEQ ID NO: 1; wherein the first nucleic acid sequence and the second nucleic acid sequence each comprise a region of sequence overlap with the other; and wherein the region of sequence overlap comprises at least about 20 contiguous nucleotides of a nucleic acid sequence corresponding to nucleotides 3598 to 3805 of SEQ ID NO: 1. Also provided are uses of AAV vector systems in the prevention or treatment of disease. [Continued on next page] WO 2017/216560 Al MIDEDIM011111 11111111OFIEHIEMI111111111111111101111111111111111111 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)) with sequence listing part of description (Rule 5.2(a))