Patent SG-11201407483Y-A (SG)
Title: Compositions and methods for modulating smn gene family expression
Inventor: KRIEG ARTHUR M
IPC: C12N15/11
Filed: 2013-05-16
Abstract: (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 21 November 2013 (21.11.2013) WIPOIPCT (10) International Publication Number WO 2013/173638 A1 (51) International Patent Classification: C12N15/11 (2006.01) C12N15/63 (2006.01) C07H21/00 (2006.01) A61K31/7088 (2006.01) (21) International Application Number: (22) International Filing Date: (25) Filing Language: (26) Publication Language: (30) Priority Data: 61/647,858 61/719,394 61/785,529 PCT/US2013/041440 16 May 2013 (16.05.2013) English English US US us 16 May 2012 (16.05.2012) 27 October 2012 (27.10.2012) 14 March 2013 (14.03.2013) (71) Applicants: RANA THERAPEUTICS, INC. [US/US]; 790 Memorial Drive, Suite 203, Cambridge, MA 02139 (US). THE GENERAL HOSPITAL CORPORATION d/b/a MASSACHUSETTS GENERAL HOSPITAL [US/US]; 55 Fruit Street, Boston, MA 02114 (US). (72) Inventors: KRIEG, Arthur M.; 173 Winding River Road, Wellesley, MA 02482 (US). SUBRAMANIAN, Romesh; 9 Alan Street, Framingham, MA 01701 (US). MCSWIG- GEN, James; 15 Beacon Street, Unit A, Arlington, MA 02474 (US). LEE, Jeannie T.; 185 Cambridge Street, Bo­ ston, MA 02114 (US). (74) Agent: YOUNG, Daniel W.; Wolf, Greenfiled & 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, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IS, JP, KE, KG, KM, KN, KP, KR, KZ, LA, LC, LK, LR, LS, LT, 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, 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, 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, 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: COMPOSITIONS AND METHODS FOR MODULATING SMN GENE FAMILY EXPRESSION 00 m i> Fuli -length no%: de!7 (90%) Fig. 1 i-H o CJ (57) Abstract: Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of SMNl or SMN2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing ex­ pression of SMNl or SMN2 that comprises exon 7. Methods for modulating expression of SMNl or SMN2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for ac­ tivating or enhancing expression of SMNl or SMN2.