Patent SG-11201808964P-A (SG)
Title: Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
Inventor: SCHNELL FREDERICK
IPC: C07H21/02
Filed: 2017-04-17
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER (19) World Intellectual Property Organization International Bureau (43) International Publication Date .....0\"\" 26 October 2017 (26.10.2017) WIPO I PCT THE PATENT COOPERATION TREATY (PCT) 1111111111101110111111111111111111111 011101110111111111110111101111111111011111 (10) International Publication Number WO 2017/184529 Al — (51) (21) (22) (25) Filing Language: (26) (30) (71) (72) International Patent Classification: (74) A61K 48/00 (2006.01) C07H 21/02 (2006.01) International Application Number: PCT/US2017/028002 (81) International Filing Date: 17 April 2017 (17.04.2017) English Publication Language: English Priority Data: 62/324,185 18 April 2016 (18.04.2016) US Applicant: SAREPTA THERAPEUTICS, INC. [US/US]; 215 First Street, Cambridge, MA 02142 (US). (84) Inventor: SCHNELL, Frederick, Joseph; 1654 NW Crest PL, Corvallis, OR 97330 (US). 85004 (US). Agent: MULHOLLAND, William F.; Snell & Wilmer LLP, One Arizona Center, 400 E. Van Buren, Phoenix, AZ 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. 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, = Title: ANTISENSE OLIGOMERS THE ACID ALPHA-GLUCOSIDASE GENE \"IT 0 as ' a ,2 .... 8 .> -5 15 = Ca c t 1, Ni cl. ' c 1 d c 0 ,0 ' 2 o 0 * A,C/ c..P„,: e,- 4,— Z 3C q l O .?. , s t.f.: cP : The present disclosure and include a targeting (GAA) gene. The least one additional nucleobase has no complementary nucleobase in the targeting sequence, and wherein the at least one additional is internal to the target region. WITH c)1: 1-1 nucleobases ---- glucosidase 0 nucleobase slk* s? ' v`k ‘.. 11 / 4 °) c ,, ,, -- • ,..,, \ - A - 4 - ,..\.! i ? ? ' 4 C1 relates sequence complementary target region N FIG. AND dr v ? METHODS 111111 5 = CU , 43\ , (1 l5 1 \"ti 'ti 0 1 '\" 4 4‘ Aki AV , tk t v y t v ._ \" l r° ,f , ,..fo . to modified antisense includes at least one additional nucleobase compared to the targeting sequence, wherein the uM 's GP s, P.,.. _ uM OF USING THE SAME FOR TREATING DISEASES ASSOCIATED = (54) = = = = = _ _ = = 15 1 '15' 15\" cl, 15' 15 * , IP 1 \"ti \", \"ti 'ti \"ti - ti N '', -' na \" .b?' P P .t.\" slx P .0 >'` ,A % . 2/ ,„'\ \" cS 0 0 0 § 3 N..... NJ. v. S' NS\" N S ' '\" N S N sf 2 4 r ° -f , .10 4c , 4. c ° -.3 c -4 c ° 1. z ..N ii ..? 1 oligonucleotides. The nucleotides described herein are of 10 to 40 to a target region within intron 1 of a pre-mRNA of the human alpha _ = = 1-1 c:7 kin 7r (57) IN at ei o [Continued on next page] WO 2017/184529 Al MIDEDIM01101 DIDIRMEM3111111111111111111111111111011111111111111111 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 amendments (Rule 48.2(h)) of receipt of