US 11,865,128 B2
Aminoglycosides and uses thereof in treating genetic disorders
Timor Baasov, Haifa (IL); Dana Atia-Glikin, Schania (IL); Jeyakumar Kandasamy, Trichy (IN); and Valery Belakhov, Haifa (IL)
Assigned to Technion Research & Development Foundation Limited, Haifa (IL)
Filed by Technion Research & Development Foundation Limited, Haifa (IL)
Filed on Apr. 8, 2021, as Appl. No. 17/226,050.
Application 15/453,990 is a division of application No. 14/866,960, filed on Sep. 27, 2015, granted, now 9,616,079, issued on Apr. 11, 2017.
Application 17/226,050 is a continuation of application No. 16/550,121, filed on Aug. 23, 2019, granted, now 10,973,839.
Application 16/550,121 is a continuation of application No. 15/914,045, filed on Mar. 7, 2018, granted, now 10,398,718, issued on Sep. 3, 2019.
Application 15/914,045 is a continuation of application No. 15/453,990, filed on Mar. 9, 2017, granted, now 9,943,533, issued on Apr. 17, 2018.
Application 14/866,960 is a continuation of application No. 14/461,477, filed on Aug. 18, 2014, granted, now 9,175,029, issued on Nov. 3, 2015.
Application 14/461,477 is a continuation of application No. 13/885,715, granted, now 8,895,519, issued on Nov. 25, 2014, previously published as PCT/IL2011/000889, filed on Nov. 17, 2011.
Claims priority of provisional application 61/414,956, filed on Nov. 18, 2010.
Prior Publication US 2021/0228608 A1, Jul. 29, 2021
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 31/7036 (2006.01); C07H 1/00 (2006.01); C07H 5/06 (2006.01); C07H 13/04 (2006.01); C07H 15/23 (2006.01); A61K 45/06 (2006.01); B65D 79/00 (2006.01)
CPC A61K 31/7036 (2013.01) [A61K 45/06 (2013.01); B65D 79/00 (2013.01); C07H 1/00 (2013.01); C07H 5/06 (2013.01); C07H 13/04 (2013.01); C07H 15/23 (2013.01)] 3 Claims
 
1. A method for treating a genetic disorder associated with a premature stop codon mutation and/or a protein truncation phenotype, the method comprising administering to a subject in need thereof a therapeutically effective amount of the compound:

OG Complex Work Unit Chemistry
or a pharmaceutically acceptable salt thereof; and
the genetic disorder being selected from the group consisting of cystic fibrosis (CF), Duchenne muscular dystrophy (DMD), ataxia-telangiectasia, Hurler syndrome, hemophilia A, hemophilia B, Usher syndrome, Tay-Sachs Becker muscular dystrophy (BMD), Congenital muscular dystrophy (CMD), Factor VII deficiency, Familial atrial fibrillation, Hailey-Hailey disease, McArdle disease, Mucopolysaccharidosis, Nephropathic cystinosis, Polycystic kidney disease, Rett syndrome, Spinal muscular atrophy (SMA), X-linked nephrogenic diabetes insipidus (XNDI) and X-linked retinitis pigmentosa.