Patent SG-11201805320X-A (SG)
Title: Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
Inventor: COWAN CHAD ALBERT
IPC: C12N15/90
Filed: 2016-12-22
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 2017/109757 A1 29 June 2017 (29.06.2017) WIPO I PCT (51) International Patent Classification: C12N15/113 (2010.01) A61K 48/00 (2006.01) C12N15/90 (2006.01) (21) International Application Number: PCT/IB2016/057958 (22) International Filing Date: 22 December 2016 (22.12.2016) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/387,424 23 December 2015 (23.12.2015) US 62/324,281 18 April 2016 (18.04.2016) US (71) Applicant: CRISPR THERAPEUTICS AG [CH/CH]; Aeschenvorstadt 36, 4051 Basel (CH). (72) Inventors: COWAN, Chad Albert; 675 West Kendall Street, Cambridge, MA 02142 (US). KABADI, Ami Meda; 675 West Kendall Street, Cambridge, MA 02142 (US). LUNDBERG, Ante Sven; 675 West Kendall Street, Cambridge, MA 02142 (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). 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)) (54) Title: MATERIALS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS AND/OR FRONTAL TEMPORAL LOBULAR DEGENERATION C90RF IVT screen 180 i> i> o\ © 1181® IP! ip 'to 1 11 p i I: : H l!i illiilillililililliiliiii! £ ilp I iilililll Sill ifllll - s. ,§5p,5 5!111 s11!141!IIts!11111!I!14!I!!I!III!11!!!!11!If o CJ o (57) : The present application provides materials and methods for treating a patient with Amyotrophic Lateral Sclerosis (ALS) and/or Frontaltemporal Lobular Degeneration (FTLD), both ex vivo and in vivo. In addition, the present application provides materials and methods for editing to modulate the expression, function or activity of the C90RF72 gene in a cell by genome editing.