Patent SG-11201909870S-A (SG)
Title: Compositions and methods of treating amyotrophic lateral sclerosis (als)
Inventor: SAH DINAH
IPC: C12N15/11
Filed: 2018-05-04
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 08 November 2018 (08.11.2018) WIPO I PCT ~ill IIIIIIIIImillion ono o Holonmoinoiliflom oimIE (10) International Publication Number WO 2018/204786 Al (51) International Patent Classification: A61K 31/713 (2006.01) Cl 2N 15/11 (2006.01) A61K 48/00 (2006.01) Cl 2N 15/113 (2010.01) A61P 25/28 (2006.01) Cl 2N 15/86 (2006.01) (21) International Application Number: PCT/US2018/031089 (22) International Filing Date: 04 May 2018 (04.05.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/501,788 05 May 2017 (05.05.2017) US 62/507,927 18 May 2017 (18.05.2017) US 62/520,100 15 June 2017 (15.06.2017) US 62/566,609 02 October 2017 (02.10.2017) US (71) Applicant: VOYAGER THERAPEUTICS, INC. [US/US]; 75 Sidney Street, Cambridge, MA 02139 (US). (72) Inventors: SAH, Dinah, Wen-Yee; 75 Sidney Street, Cam- bridge, MA 02139 (US). CHEN, Qingmin; 75 Sidney Street, Cambridge, MA 02139 (US). HOU, Jinzhao; 75 Sidney Street, Cambridge, MA 02139 (US). 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)) Agent: WARD, Donna T.; DT Ward, PC, 142A Main Street, Groton, MA 01824 (US). 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, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, = (54) Title: COMPOSITIONS AND METHODS OF TREATING AMYOTROPHIC LATERAL SCLEROSIS (ALS) 00 N O 00 O O (57) : The present invention relates to adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating amyotrophic lateral sclerosis (ALS). The present invention relates to compositions, methods and processes for the design, preparation, manufacture, use and/or formulation of AAV particles comprising modulatory polynucleotides, e.g., polynucleotides en- codin g at least one small interfering RNA (siRNA) molecules which target the superoxide dismutase 1 (SOD1) gene. Methods for using the AAV particles to inhibit the expression of the SOD1 gene in a subject with a neurodegenerative disease (e.g., amyotrophic lateral sclerosis (ALS)) are also disclosed. 130 110 120