Patent SG-11201901265S-A (SG)
Title: Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways
Inventor: ANDERSON DAVID W
IPC: C12Q1/68
Filed: 2016-07-21
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 11111111011110111010101111101011111111111101111001111111111011111101111011111 (43) International Bureau 0.. .1 j International Publication Date ..... ..,,/ (10) International Publication Number WO 2017/015491 Al 26 January 2017 (26.01.2017) WIPO I PCT (51) International Patent Classification: (72) Inventors: ANDERSON, David W.; 3911 Rockwood C12N 15/09 (2006.01) A61P 25/28 (2006.01) Farm Road., Newtown Square, Pennsylvania 19073 (US). C12N 15/10 (2006.01) C12Q 1/68 (2006.01) SCHNEIDER, Jay S.; 1665 Blue Jay Lane, Cherry Hill, C12N 15/113 (2010.01) A61K 35/30 (2015.01) New Jersey 08003 (US). C12N 15/12 (2006.01) (74) Agent: VOS STRACHE, Kyle; Cozen O'Connor, One (21) International Application Number: Liberty Place, 1650 Market Street, Philadelphia, PCT/US2016/043393 Pennsylvania 19103 (US). (22) International Filing Date: (81) Designated States (unless otherwise indicated, for every 21 July 2016 (21.07.2016) kind of national protection available): AE, AG, AL, AM, (25) Filing Language: English AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DK, DM, (26) Publication Language: English DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, (30) Priority Data: KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, 62/194,954 21 July 2015 (21.07.2015) US MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, 62/194,910 21 July 2015 (21.07.2015) US PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, (71) Applicant: THOMAS JEFFERSON UNIVERSITY SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, [US/US]; Innovation Pillar, 901 Walnut Street, Phil- TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. adelphia, Pennsylvania 19107 (US). [Continued on next page] (54) Title: GENE THERAPIES FOR NEURODEGENERATWE DISORDERS TARGETING GANGLIOSIDE BIOSYNTHETIC PATHWAYS (57) : A composition of matter comprising an ad- FIG. lA eno-associated virus (AAV) or other human compatible vir- us, encoding the gene for Sialidase Neu3, B3Galt4, St3Ga12, gal or combinations thereof, and a neuron specific promoter, tasial wherein the composition is suitable for administration to a Fr patient comprising injecting the AAV or other human com- L Dctc.sylcci onlific sia3 patible virus into the brain by intracranial stereotaxic injunc - II. tion; wherein the AAV's encoding for the Sialidase Neu3, ,111 B3Galt4, St3Ga12, or combinations thereof enhance and/or normalize levels of GM1 in neurons, providing both thera- 4gaIntl peutic relief and disease modifying effects in specific areas of the brain relevant to particular neurodegenerative dis- 1 eases. I sl , 1-1 11 C:r 7r ir) 11 0 IN 11 0 ei C WO 2017/015491 Al MIDEDIMOMOIDEIRMEMONIMONIEHOMOVOIMIE (84) Designated States (unless otherwise indicated, for every Declarations under Rule 4.17: kind of regional protection available): ARIPO (BW, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, GH, SZ, of inventorship (Rule 4.17(iv)) Published: TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, DE, LT, SE, — with international search report (Art. 21(3)) before the expiration of the time limit for amending the SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). GA, claims and to be republished in the event amendments (Rule 48.2(h)) of receipt of