BOPI 01 BR/2024 REPERTOIRE NUMERIQUE 14 Fig. 1a & Fig.1b ________________________________________ (11) 21335 Consulter le mémoire (51) A61K 31/4045 (2018.01); A61K 45/06 (2018.01); A61K 9/00 (2018.01) ; A61P 13/12 (2018.01) (21) 1202200361 - PCT/US2021/021037 (22) 05/03/2021 (30) US n° 62/986,096 du 06/03/2020 (54) Methods of treating APOL-1 dependent focal segmental glomerulosclerosis. (72) EGBUNA, Ifeatu (US); HARE, Brian J. (US); KRUG, Alexander Wolfgang (US); MALLALIEU, Navita (US) et WU, Shu-Pei (US) (73) Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, BOSTON, Massachusetts 02210 (US) (74) SPOOR & FISHER Inc. NGWAFOR & PARTNERS SARL, The House of Gideon, Golf/Bastos Quarter, Opposite The American Embassy, Entrance-St. John Paul II Boulevard, P.O. Box 8211, YAOUNDE (CM). (57) This application describes methods of inhibiting APOL1 and treating APOL1-mediated diseases comprising administering Compound I and/or a pharmaceutically acceptable salt thereof. Compound I The application also describes pharmaceutical compositions comprising Compound I and/or pharmaceutically acceptable salt thereof. Compound I & Fig. 1 ________________________________________ (11) 21336 Consulter le mémoire (51) C12N 15/113 (2018.01) (21) 1202200362 - PCT/US2021/021199 (22) 05/03/2021 (30) US n° 62/986,150 du 06/03/2020 ; US n° 63/109,196 du 03/11/2020 (54) Modified short interfering nucleic acid (siNA) molecules and uses thereof. (72) BEIGELMAN, Leonid (US); RAJWANSHI, Vivek Kumar (US); HONG, Jin (US); PANDEY, Rajendra K. (US); HOSSBACH, Markus (DE); ELTE PU, Lax man (US); MONTERO, Saul Martinez (US) et DE COSTA, N. Tilani S. (US) (73) ALIGOS THERAPEUTICS, INC., 1 Corporate Drive, 2nd Floor, South San Francisco, California 94080 (US) (74) SPOOR & FISHER Inc. NGWAFOR & PARTNERS SARL, The House of Gideon, Golf/Bastos Quarter, Opposite The American Embassy, Entrance-St. John Paul II Boulevard, P.O. Box 8211, YAOUNDE (CM). (57) Disclosed herein are short interfering nucleic acid (siNA) molecules comprising modified nucleotides and uses therof. The siNA molecules may be double stranded and comprise modified nucleotides selected from 2’-O-methyl nucleotides and 2’-fluoro nucleotides. Further disclosed herein are siNA molecules comprising (a) a phosphorylation blocker, conjugated moiety, or 5’-
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