Patent SG-11201806663T-A (SG)
Title: Closed-ended linear duplex dna for non-viral gene transfer
Inventor: KOTIN ROBERT
IPC: C12N15/66
Filed: 2017-03-03
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (10) International Publication Number (43) International Publication Date WO 2017/152149 Al 8 September 2017 (08.09.2017) WIPO I PCT 111111111111110111011111111111010111110111011111111011101011111110111111111110111111 (51) International Patent Classification: A61K 48/00 (2006.01) C12N 15/64 (2006.01) C12N 15/09 (2006.01) C12N 15/66 (2006.01) (21) International Application Number: PCT/US2017/020828 (22) International Filing Date: 3 March 2017 (03.03.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/303,047 3 March 2016 (03.03.2016) US 62/394,720 14 September 2016 (14.09.2016) US 62/406,913 11 October 2016 (11.10.2016) US (71) Applicant: UNIVERSITY OF MASSACHUSETTS [US/US]; 225 Franklin Street, Boston, MA 02110 (US). (72) Inventors: KOTIN, Robert, M.; 6510 Broxburn Dr., Bethesda, MD 20817 (US). CECCHINI, Sylvain; 67 Adams St., Westborough, MA 01581 (US). (74) Agent: YOUNG, Daniel, W.; Wolf, Greenfield & Sacks, P.C., 600 Atlantic Avenue, Boston, MA 02210-2206 (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, 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, 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)) - (54) Title: CLOSED-ENDED LINEAR DUPLEX DNA FOR NON-VIRAL GENE TRANSFER G — C G — C T — A C — G G— C C' C — G C — G C — G 3'AACCGG7GAGGGAGAGACGCGCGACCGAGCGAGTGACTCCGC • C 1111111111111111 1111 11111111111111111111' T 5 'AGGAACCCTAGEGATGGAGTTGGCCACTCCCTCTC7GCGCGC7CGCTCGCTCACTGAGGCC SEQ ID NO: 8 D. trs RBE A C B C FIG. 1A (57) : Aspects of the disclosure relate to a nucleic acid comprising a heterologous nucleic acid insert flanked by interrupted self-complementary sequences, wherein one self-complementary sequence is interrupted by a cross-arm sequence forming two op - posing, lengthwise- symmetric stem-loops, and wherein the other of the self-complementary sequences is interrupted by a truncated cross-arm sequence. Methods of delivering the nucleic acid to a cell are also provided. W O 20 17 / 15 2149 Al G — C C — G A A A • G — C — G — G — G G — C G — C G C G — C G — C C • G • T C • G G — C C — G C — G B ' C — G G — C G — C G — C A A A SEQ ID NO: 24