Patent SG-11201809468X-A (SG)
Title: Oligonucleotide analogues targeting human lmna
Inventor: ERDOS MICHAEL R
IPC: C12N15/113
Filed: 2017-04-28
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 11111111111111111111111111111111111111111111111111111111111111111111111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2017/190041 Al 02 November 2017 (02.11.2017) W I PO I PCT (51) International Patent Classification: 0133 Cole Student Activities Building, College Park, Mary- C12N 15/113 (2010.01) C07H 21/00 (2006.01) land 20742 (US). A61K 31/712 (2006.01) (72) Inventors: ERDOS, Michael R.; Office of Technology (21) International Application Number: Transfer, NIH, 6011 Executive Boulevard, Suite 325, MSC PCT/US2017/030174 7660, Bethesda, Maryland 20892 (US). COLLINS, Fran- cis S.; Office of Technology Transfer, NIH, 6011 Exec- (22) International Filing Date: utive Boulevard, Suite 325, MSC 7660, Bethesda, Mary- 28 April 2017 (28.04.2017) land 20892 (US). CAO, Kan; 2215 Davit Court, Bowie, (25) Filing Language: English Maryland 20721 (US). KOLE, Ryszard; 136 Southwest (26) Publication Language: English Washington Avenue #405, Corvallis, Oregon 97333 (US). BESTWICK, Richard Keith; 3905 NW Clarence Circle, (30) Priority Data: Corvallis, Oregon 97330 (US). 62/330,027 29 April 2016 (29.04.2016) US (74) Agent: SUN, Eileen S. et al.; Seed Intellectual Property (71) Applicants: SAREPTA THERAPEUTICS, INC. Law Group LLP, Suite 5400, 701 Fifth Avenue, Seattle, [US/US]; 215 First Street, Suite 415, Cambridge, Mass- Washington 98104-7064 (US). achusetts 02142 (US). THE UNITED STATES OF (81) Designated States (unless otherwise indicated, for every AMERICA, AS REPRESENTED BY THE SEC- kind of national protection available): AE, AG, AL, AM, RETARY, DEPT. OF HEALTH AND HUMAN AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, SERVICES [US/US]; Office of Technology Transfer, NIH, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, 6011 Executive Boulevard, Suite 325, MSC 7660, Bethes- DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, da, Maryland 20892 (US). UNIVERSITY OF MARY- HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, LAND [[JS/US]; Office of Technology Commercialization, 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, = (54) Title: OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA = ddPCR assay 12-week PPMO pilot study: heart 250 = = 200 E Progerin copies/p1 B Wt LMNA copies/pi 1 2 Progerin/LMNA (%) = = = = 150 = 0) 2 = 0_ < 1 0 4 ,, 7 4 ior /e = z _ 50 11 ,—, ii. 0 / /• /7 ''''' 0 '< • — / / % % %. . , er fr ?;.... 4,... ... 11 Or d r: 0. ,,,.. 0 1 0,11 C5) 2 0 CONTROL PPMO1 PPMO1 PPMO1 PPMO2 PPMO2 PPMO2 0 60 mg/kg 20 mg/kg 7 mg/kg 60 mg/kg 20 mg/kg 7 mg/kg 01 ,-1 FIG. 1 IN ,-1 © (57) : Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced f'1 LMNA mRNA isoforms that encode progerin. o [Continued on next page] WO 2017/190041 Al IIMEDIMOMOIDEIROIDEMOMOHMONNIVOIIIMEMOIMIE 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). Declarations under Rule 4.17: — as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) 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))