Technion Research & Development Foundation Ltd

Eloxx Pharmaceuticals Ltd.

The Scripps Research Institute

Timor Baasov

Valery Belakhov

Jeyakumar Kandasamy

Dana Atia-Glikin

Mariana Hainrichson

Igor Nudelman

Tamar Ben-Yosef

Annie Rebibo-Sabbah

Dalia Shallom-Shezifi

Shmuel Tuvia

Chi-Huey Wong

Zhiyuan Zhang

Ian Ollmann

Xin-Shan Ye

Varvara Pokrovskaya

Micha Fridman

Boris Smolkin

Alina Khononov

Michal Shavit Kishkober

Dori Pelled

Sima Yaron

Modified aminoglycoside compounds represented by Formula I as defined and described in the specification are provided. The modified aminoglycosides feature a diamine-containing functional moiety at one or more of positions 3', 4′ and 6′. Uses of the modified aminoglycosides as antimicrobial (e.g., antibacterial) agents, and in treating medical conditions associated with microorganisms, are also provided.

Modified aminoglycoside compounds and uses thereof in disabling bacterial ribosome

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

Aminoglycosides and uses thereof in treating genetic disorders

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.

Aminoglycoside derivatives and uses thereof in treating genetic disorders

Novel aminoglycosides, represented by Formulae Ia and Ib, 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-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.

A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5&#x2033; 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: or a pharmaceutically acceptable salt thereof, wherein Ris selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.

Compounds, methods and uses of pseudo-trisaccharide aminoglycosides represented by the general formula I: or a pharmaceutically acceptable salt thereof, wherein Ris selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification, in the treatment of Rett syndrome are disclosed.

Use of aminoglycoside analogs in the treatment of Rett syndrome

A new class of paromomycin-derived aminoglycosides, which exhibit efficient stop-codon mutation suppression activity, low toxicity and high selectivity towards eukaryotic cells are provided. Also provided are chemical and chemo-enzymatic processes of preparing these paromomycin-derived aminoglycosides and intermediates thereof, as well as pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders.

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Haifa, Israel

Timor Baasov