A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5 position, exhibiting efficient stop codon mutation readthrough activity, low cytotoxicity and high selectivity towards eukaryotic translation systems are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders, as well as processes of preparing these aminoglycosides. The disclosed aminoglycosides can be represented by the general formula I: ##STR00001##
or a pharmaceutically acceptable salt thereof, wherein R.sub.1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.

Novel pseudo-trisaccharide aminoglycosides, represented by Formula I, 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.

Novel pseudo-trisaccharide aminoglycosides, represented by Formula I, 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.

A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5 position, exhibiting efficient stop codon mutation readthrough activity, low cytotoxicity and high selectivity towards eukaryotic translation systems are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders, as well as processes of preparing these aminoglycosides. The disclosed aminoglycosides can be represented by the general formula I:

##STR00001## or a pharmaceutically acceptable salt thereof, wherein R.sub.1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.

A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5 position, exhibiting efficient stop codon mutation readthrough activity, low cytotoxicity and high selectivity towards eukaryotic translation systems are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders, as well as processes of preparing these aminoglycosides. The disclosed aminoglycosides can be represented by the general formula I:

##STR00001## or a pharmaceutically acceptable salt thereof, wherein R.sub.1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.

Synthetic pathways for preparing pseudo-trisaccharide aminoglycoside compounds represented by Formula I or Ia as defined in the specification and donor and acceptor compounds useful for preparing such compounds are provided. A process of stereoselectively preparing compounds represented by Formula III as defined in the specification, while avoiding chromatographic separation of stereoisomers are also provided. Compounds prepared by the described processes and uses thereof are also provided.

Synthetic pathways for preparing pseudo-trisaccharide aminoglycoside compounds represented by Formula I or Ia as defined in the specification and donor and acceptor compounds useful for preparing such compounds are provided. A process of stereoselectively preparing compounds represented by Formula III as defined in the specification, while avoiding chromatographic separation of stereoisomers are also provided. Compounds prepared by the described processes and uses thereof are also provided.

A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5 position, exhibiting efficient stop codon mutation readthrough activity, low cytotoxicity and high selectivity towards eukaryotic translation systems are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders, as well as processes of preparing these aminoglycosides. The disclosed aminoglycosides can be represented by the general formula I: ##STR00001##
or a pharmaceutically acceptable salt thereof, wherein R.sub.1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.

A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5 position, exhibiting efficient stop codon mutation readthrough activity, low cytotoxicity and high selectivity towards eukaryotic translation systems are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders, as well as processes of preparing these aminoglycosides. The disclosed aminoglycosides can be represented by the general formula I: ##STR00001##
or a pharmaceutically acceptable salt thereof, wherein R.sub.1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.

A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5 position, exhibiting efficient stop codon mutation readthrough activity, low cytotoxicity and high selectivity towards eukaryotic translation systems are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders, as well as processes of preparing these aminoglycosides. The disclosed aminoglycosides can be represented by the general formula I:

##STR00001##

or a pharmaceutically acceptable salt thereof, wherein R.sub.1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.