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RiboStruct is developing the first generic treatment for genetic diseases.

RiboStruct leverages the long-term work of Marat Yusupov and Gulnara Yusupova on the crystallographic study of the eukaryotic ribosome to develop new drugs, in particular "readthrough compounds" targeting the human ribosome for the treatment of monogenic genetic diseases when they are caused by nonsense mutations (10-15% of cases).
Distinctively from gene therapies, where a specific program is needed for each disease, RiboStruct develops a pharmacological "protein repair therapy" approach, with the goal to cure multiple diseases with the same molecule by acting on a common mechanism. More than 7000 genetic diseases have been described so far, affecting altogether 300 million people worlwide.

Genetic diseases

Nonsense mutations

Premature stop codons

Readthrough

Human ribosome

Structure-based drug design

Dr Renaud Jean-Paul, President & CEO-CSO

RiboStruct is trying to achieve a first round of funding.

Aminoglycosides are known to help the ribosome overcome premature stop codons to restore the synthesis of full-length proteins but their high toxicity prevents their use on the long term as needed for the treatment of monogenic diseases caused by nonsense mutations.

RiboStruct is developing non-aminoglycoside readthrough compounds using a fragment- and structure-based drug design approach relying on an exclusive know-how on the crystallographic structure determination of the whole eukaryotic ribosome in complex with ligands and on the strong biophysical capabilities of its strategic partner NovAliX (http://www.novalix-pharma.com/).