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1. (WO2017037717) AMINOGLYCOSIDE DERIVATIVES AND USES THEREOF IN TREATING GENETIC DISORDERS

Pub. No.:    WO/2017/037717    International Application No.:    PCT/IL2016/050965
Publication Date: Fri Mar 10 00:59:59 CET 2017 International Filing Date: Sat Sep 03 01:59:59 CEST 2016
IPC: C07H 15/23
A61K 31/7036
A61P 7/04
A61P 21/04
C07H 15/222
C07H 15/224
Applicants: ELOXX PHARMACEUTICALS LTD.
Inventors: BAASOV, Timor
Title: AMINOGLYCOSIDE DERIVATIVES AND USES THEREOF IN TREATING GENETIC DISORDERS
Abstract:
Novel pseudo-disaccharide and pseudo-trisaccharide aminoglycosides, represented by Formulae I or Ia, as defined in the instant specification, designed to exhibit stop codon mutation readthrough activity, are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic diseases and disorders, such as diseases and disorders associated with stop codon mutations.