BOPI 02 BR/2023 REPERTOIRE NUMERIQUE 31 (74) Cabinet ISIS CONSEILS (SCP), Bastos Rue 1.862 B.P. 15067, YAOUNDE (CM). (57) The present invention describes a novel bacterial nuclease of the CRISPR-Cas9 system from the bacterium P. pneumotropica, as well as the use thereof to form strictly specific double-strand breaks in a DNA molecule. This nuclease has unusual properties and may be used as a tool for modifying the genomic DNA sequence in the cell of a unicellular organism or a multicellular organism. Thus, the versatility of the available CRISPR-Cas9 systems is increased, which fact will enable the use of various variants of Cas9 nucleases for cutting genomic or plasmid DNA in various organisms, in a larger number of specific sites and/or under various conditions. Fig. 11 Consulter le mémoire ________________________________________ (11) 20813 (51) A61K 31/4439 (2018.01); A61P 7/00 (2018.01) (21) 1202200189 - PCT/US2020/060923 (22) 17/11/2020 (30) US n° 62/937,706 du 19/11/2019; US n° 62/940,154 du 25/11/2019 (54) Methods of administering voxelotor. (72) WASHINGTON, Caria B. (US) (73) GLOBAL BLOOD THERAPEUTICS, INC., 181 Oyster Point Boulevard, SOUTH SAN FRANCISCO, California 94080 (US) (74) S.C.P AKKUM, AKKUM & Associates, No. 1777, Rue 6.261, Yaoundé, Auditorium Jean Paul II, Quartier Mbankolo, B.P. 4966, YAOUNDE (CM). (57) Provided herein are methods of using voxelotor for the treatment of sickle cell disease in patients also having severe hepatic impairment. Also provided herein are methods for administering voxelotor and avoiding or lessening adverse drug interactions with a CYP3A4 inducer or inhibitor. Also provided herein are methods for avoiding interactions with voxelotor, a moderate CYP3A4 inhibitor.
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