Patent SG-11201811189R-A (SG)
Title: Variant adeno-associated viruses and methods of using
Inventor: DUDMAN JOSHUA
IPC: C12N7/01
Filed: 2017-06-15
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1111111011110111011111111010111110011111111111111111111111111111111111111111111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\"\" WO 2017/218842 Al 21 December 2017 (21.12.2017) WIP0 I PCT (51) International Patent Classification: Kimberly [US/US]; 19700 Helix Drive, Ashburn, Vir- C07K 14/015 (2006.01) A61K 48/00 (2006.01) ginia 20147 (US). SCHAFFER, David [US/US]; 19 Rima C12N 7/01 (2006.01) Court, Danville, California 94526 (US). TERVO, Gowan (21) International Application Number: [CA/US]; 19700 Helix Dr., Ashburn, Virginia 20147 (US). PCT/US2017/037781 VISWANATHAN, Sarada [US/US]; 19700 Helix Drive, Ashburn, Virginia 20147 (US). (22) International Filing Date: (74) Agent: PARSONS, Angela M. et al.; Fish & Richardson 15 June 2017 (15.06.2017) P.C., P.O. Box 1022, Minneapolis, Minnesota 55440-1022 (25) Filing Language: English (US). (26) Publication Language: English (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, (30) Priority Data: AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, 62/350,361 15 June 2016 (15.06.2016) US CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, 62/404,585 05 October 2016 (05.10.2016) US DZ, EC, EE, EG, ES, FL GB, GD, GE, GH, GM, GT, HN, (71) Applicants: HWANG, Bum-Yeol [KR/US]; 303 Deer- HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, field Dr., Moraga, California 94556 (US). KARPOVA, Al- KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, la [US/US]; 19700 Helix Drive, Ashburn, Virginia 20147 MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, (US). 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, (72) Inventors; and TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. _ (71) Applicants: DUDMAN, Joshua [US/US]; 207 Browns = Meadow Ct, Leesburg, Virginia 20176 (US). HANT- (84) Designated States (unless otherwise indicated, for every MAN, Adam [US/US]; 19700 Helix Drive, Ashburn, Vir- kind of regional protection available): ARIPO (BW, GH, = ginia 20147 (US). LOOGER, Loren [US/US]; 20149 GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, _ Broad Run Dr, Sterling, Virginia 20165 (US). RITOLA, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, = = (54) Title: VARIANT ADENO-ASSOCIATED - rAAVX or CAV2 = Syn-Cre = = VIRUSES AND METHODS OF USING (57) : The present disclosure provides AAV variants that exhibit a pref- erence for retrograde movement in neurons and methods of using such variants. I I = = tx : = = Rosa26-LSL-H2B-GFP = = = = = 11 ei ii- cc cc 1-1 ei IN 1-1 FIG. 2B 0 ei 4 .BPN C [Continued on next page] WO 2017/218842 Al MIDEDIMOMOIDEIRMEM00111HEIEHIMOVOIMIE 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))