US 11,773,393 B2
Treatment of liver diseases with cell death inducing DFFA like effector B (CIDEB) inhibitors
Niek Verweij, Tarrytown, NY (US); Luca Andrea Lotta, Tarrytown, NY (US); Aris Baras, Tarrytown, NY (US); Mary Haas, Tarrytown, NY (US); Jonas Nielsen, Tarrytown, NY (US); Olukayode Sosina, Tarrytown, NY (US); and Adam Locke, Tarrytown, NY (US)
Assigned to Regeneron Pharmaceuticals, Inc., Tarrytown, NY (US)
Filed by Regeneron Pharmaceuticals, Inc., Tarrytown, NY (US)
Filed on Mar. 31, 2022, as Appl. No. 17/709,705.
Application 17/709,705 is a continuation of application No. 17/560,008, filed on Dec. 22, 2021.
Claims priority of provisional application 63/257,137, filed on Oct. 19, 2021.
Claims priority of provisional application 63/246,101, filed on Sep. 20, 2021.
Claims priority of provisional application 63/149,258, filed on Feb. 13, 2021.
Claims priority of provisional application 63/129,725, filed on Dec. 23, 2020.
Prior Publication US 2022/0282253 A1, Sep. 8, 2022
This patent is subject to a terminal disclaimer.
Int. Cl. C12N 15/113 (2010.01); A61P 1/16 (2006.01); A61K 31/7088 (2006.01); A61K 38/46 (2006.01); C12Q 1/6883 (2018.01)
CPC C12N 15/113 (2013.01) [A61K 31/7088 (2013.01); A61K 38/465 (2013.01); A61P 1/16 (2018.01); C12N 15/1137 (2013.01); C12Q 1/6883 (2013.01); C12N 2310/122 (2013.01); C12N 2310/14 (2013.01); C12N 2310/531 (2013.01); C12N 2320/31 (2013.01); C12Q 2600/106 (2013.01); C12Q 2600/158 (2013.01)] 27 Claims
 
1. A method of treating a subject with a cell death inducing DFFA like effector B (CIDEB) inhibitor, wherein the subject has a liver disease or is at risk of developing a liver disease, the method comprising the steps of:
determining whether the subject has a CIDEB variant nucleic acid molecule by:
obtaining or having obtained a biological sample from the subject; and
performing or having performed a sequence analysis on the biological sample to determine if the subject has a genotype comprising the CIDEB variant nucleic acid molecule, wherein the CIDEB variant nucleic acid molecule is a missense variant, a splice-site variant, a stop-gain variant, a start-loss variant, a stop-loss variant, a frameshift variant, or an in-frame indel variant, or a variant that encodes a truncated CIDEB polypeptide; and
administering or continuing to administer the CIDEB inhibitor in a standard dosage amount to a CIDEB reference subject; and
administering or continuing to administer the CIDEB inhibitor in a dosage amount that is the same as or less than a standard dosage amount to a subject that is heterozygous for the CIDEB variant nucleic acid molecule;
wherein the presence of a genotype having the CIDEB variant nucleic acid molecule indicates the subject has a decreased risk of developing the liver disease or has a decreased risk of developing a more severe form of the liver disease.