Patent SG-11201804443U-A (SG)
Title: Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
Inventor: AZNAREZ ISABEL
IPC: C12Q1/68
Filed: 2016-12-14
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -' Organization International Bureau (43) International Publication Date ..... .....r .,„0 22 June 2017(22.06.2017) WIPO I PCT (10) WO International Publication Number 111111111111311111111111111111111111111111111111111111111111111111111111111111111111111111 2017/106377 Al (51) International Patent Classification: AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, C12N 15/113 (2010.01) C12Q 1/68 (2006.01) BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, C12N 15/11 (2006.01) 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, (21) International Application Number: KP, KR, KW, KZ, LA, LC, LK, LR, LS LU, LY MA, PCT/US2016/066708 MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (22) International Filing Date: NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, 14 December 2016 (14.12.2016) 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, (25) Filing Language: English ZA, ZM, ZW. (26) Publication Language: English (84) Designated States (unless otherwise indicated, for every (30) Priority Data: kind of regional protection available): ARIPO (BW, GH, 62/267,251 14 December 2015 (14.12.2015) US GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, (71) Applicants: COLD SPRING HARBOR LABORAT- TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, ORY [US/US]; One Bungtown Road, Nicholas Buliding, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, P.O. Box 100, Cold Spring Harbor, New York 11724 (US). LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, STOKE THERAPEUTICS, INC. [US/US]; 3 Preston SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, Court, Bedford, Massachusetts 01730 (US). GW, KM, ML, MR, NE, SN, TD, TG). (72) Inventors: AZNAREZ, Isabel; 55 Magazine Street, Apt. Published: 12A, Cambridge, Massachusetts 02139 (US). NASH, Huw with international search report (Art 21(3)) M.; 4 Washington St., Lexington, Massachusetts 02421 — before the expiration of the time limit for amending the (US). claims and to be republished in the event of receipt of (74) Agent: SCHULTZ, Jason B.; Wilson Sonsini Goodrich & amendments (Rule 48.2(h)) Rosati, 650 Page Mill Road, Palo Alto, California 94304 — with sequence listing part of description (Rule 5.2(a)) (US). = (81) Designated States (unless otherwise indicated, for every kind of national available): AE, AG, AL, AM, protection (54) Title: ANTISENSE OLIGOMERS FOR TREATMENT OF AUTOSOMAL DOMINANT MENTAL RETARDATION-5 AND DRAVET SYNDROME IN TARGET REGIONS RIC pre-mRNA f 74 7 ,7 er/ war/ P inton retar ed 1-1 .4t IN ef) r------i 10 1 / 46 1 1 i CA 6 grtaagninmInnnrmnrmnrmrmnnnrnfinrmminminmmrm spliuz n y g 0 1-1 FIG. 1 IN Il O (57) : Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.