Patent CA-3100006-A1 (CA)
Inventor: VANDENBERGHE LUK H
IPC: A61K35/76
Filed: 2019-05-10
Abstract: (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY  (PCT) (19) World Intellectual Property                        1111 M EMMEll Organization                                 HI International Bureau                             (10) International Publication Number (43) International Publication Date                WO 2019/217911 Al 14 November 2019 (14.11.2019) %NIP() I PCT (51) International Patent Classification:    setts 02114 (US). ZINN, Eric; 11 Manning Road, Lynn, A6IK 35/76 (2006.01)    C12N 5/00 (2006.01)    Massachusetts 01902 (US). (21) International Application Number:                                         (74) Agent: PARSONS, M. Angela; Fish & Richardson P.C., PCT/US2019/031851    P.O. Box 1022, Minneapolis, Minnesota 55440 (US). (22) International Filing Date:                                                (81) Designated States (unless otherwise indicated, for every 10 May 2019 (10.05.2019)    kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, (25) Filing Language:                   English CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, (26) Publication Language:              English    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, (30) Priority Data: KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, 62/670,543                        11 May 2018 (11.05.2018)    US MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, 62/841,179                        30 April 2019 (30.04.2019)    US OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, (71) Applicants: MASSACHUSETTS EYE AND EAR    SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, INFIRMARY [US/US]; 243 Charles Street, Boston,    TR, Tr, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. Massachusetts 02114 (US). THE SCHEPENS EYE (84) Designated States (unless otherwise indicated, for every RESEARCH INSTITUTE, INC. [US/US]; 20 Staniford kind of regional protection available): ARIPO (BW, GH, Street, Boston, Massachusetts 02114 (US). GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, (72) Inventors: VANDENBERGITE, Luk H.; 14 Indian Hill    UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, Road, Weston, Massachusetts 02493 (US). SCHMIT,    TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, Pauline; 1167 Boylston Street, Apt. 36, Boston, Massachu-    EE, ES, FI, FR, GB, GR, HR, HU, 1E, IS, IT, LT, LU, LV, setts 02215 (US). TIPPER, Christopher; 6 Thingvalla Av-    MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, enue, Cambridge, Massachusetts 02138 (US).UNZU, Car-    TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, men; 4 Longfellow Place, Apt. 1710, Boston, Massachu-    KM, ML, MR, NE, SN, TD, TG). = (54) Title: LIVER-SPECIFIC TROPISM OF ADENO-ASSOCIATED VIRUSES (57) Abstract: This disclosure provides compositions and methods for altering or changing the tissue tropism, e.g., liver tropism, of ade-100   _________________________________ no-associated viruses (AAV). g10  ______________________________ 0.1 001  ________________ 0.001  ____________________________ Anc80L65                   Anc80L65 G 266A    UHnjected Toggle ON             Toggle OFF p3 G                  p3 = A Code X1 X2 X3 X4 X5 X6 X7 X8 89 X10 X11 OK A AK Q I TNQ AN  1 SUR t E A X b S Anc80L65 R ACK Q T T N E A D Anc0OL65 G266AR    A    A    K    Q    T    T    NE    A    D FIG. 11 [Continued on next page] Date recue/Date Received 2020-1 1-1 1 Published: ¨    with international search report (Art. 21(3)) Date reçue/Date Received 2020-1 1-1 1