Patent SG-11201806605R-A (SG)
Title: Regulation of gene expression through aptamer-modulated polyadenylation
Inventor: VOLLES MICHAEL
IPC: C12N15/86
Filed: 2017-02-02
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 2017/136591 Al 10 August 2017 (10.08.2017) WIPO I PCT 111111 11111111 0 111011111111111 010 11111 01110111 0111111111111111101111111111111 0111111 (51) International Patent Classification: A61K 48/00 (2006.01) CO7H 21/04 (2006.01) C12N 15/86 (2006.01) (21) International Application Number: PCT/US2017/016279 (22) International Filing Date: 2 February 2017 (02.02.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/290,200 2 February 2016 (02.02.2016) US (71) Applicants: MEIRAGTX UK II LIMITED [GB/GB]; 92 Britannia Walk, London, N1 7NQ (GB). MEIRAGTX, LLC [US/US]; 450 East 29th Street 15th Floor, New York, NY 10016 (US). (72) Inventors: VOLLES, Michael, J.; 69 Bolton Street Apt. 102, Cambridge, MA 02140 (US). DANOS, Olivier, F.; 49 Hancock Street, Boston, MA 02114 (US). GUO, Xuecui; 1 Bungtown Road, Cold Spring Harbor, NY 11724 (US). (74) Agents: SOMERVILLE, Deborah, A. et al.; Fox Roth- schild, LLP, Princeton Pike Corporate Center, 997 Lenox Drive, Building 3, Lawrenceville, NJ 08648-2311 (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). arations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.1700) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) ished: 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)) Decl Publ W O 20 17 / 1365 9 1 Al (54) Title: REGULATION OF GENE EXPRESSION THROUGH APTAMER-MODULATED POLYADENYLATION (57) : The invention provides polynucleotide constructs for the regulation of gene expression by aptamer-based modulation of Ul small nuclear ribonucleoprotein (snRNP)-mediated suppression of polyadenylation and methods of using the constructs to regulate gene expression in response to the presence or absence of a ligand that binds the aptamer. The polynucleotide construct con - tains a Ul binding site in the context of a riboswitch comprising an effector region and an aptamer such that when the aptamer binds a ligand, target gene expression occurs.