Developing treatments for genetic diseases and cancers caused by nonsense mutations.

Developing treatments for genetic diseases and cancers caused by nonsense mutations

Monogenic diseases are all rare diseases but, collectively, they

affect 300 million people worldwide.

More than 7,000 monogenic diseases have already been described.

In each case, a single gene defect impairs in various ways the production of the corresponding protein in its fully functional state, causing the disease.

The therapeutic need is huge but there are at present almost no curative treatments.

One way in which protein production can be affected arises from

nonsense mutations (10-15% of patients in average)

that lead to the loss of the full-length protein through different mechanisms. The ability of specific molecules to restore the synthesis of a full-length protein through a mechanism called

READTHROUGH is a new therapeutic approach for such cases.

Unlike gene therapies that target a single defective gene each, the READTHROUGH approach could be applied in the treatment of numerous genetic diseases since it bypasses the deleterious mutation through a gene-independent mechanism at the level of the human ribosome, the protein factory in all our cells.

Urania therapeutics exploits a unique know-how in ribosome X-ray crystallography and cryo-EM to develop safe and efficient READTHROUGH compounds through structure-based drug design.

About Us

Urania Therapeutics (formerly RiboStruct) was created end of 2015 as a spinoff of IGBMC to leverage the expertise of Marat Yusupov & Gulnara Yusupova in the field of ribosome crystallography and cryo-EM to develop new drugs.

Urania is developing readthrough compounds targeting the human ribosome

following a structure-based drug design approach for the treatment of genetic diseases and some cancers caused by nonsense mutations.


Urania Therapeutics provides a therapeutic solution for the

treatment of genetic diseases

caused by nonsense mutations (10-15% of cases), which introduce a STOP codon into the coding sequence of the gene, leading to the premature termination of protein synthesis at the level of of the ribosome and therefore to the production of a non-functional truncated protein at the origin of the disease.

Our investors

Our investors share our vision of a future where patients with no therapeutic options at present get treated with readthrough compounds.



In collaboration with the Yusupov lab at IGBMC, Urania publishes today an article in Nature entitled "Accuracy mechanism of eukaryotic ribosome translocation" describing the 3.2-Å-resolution X-ray structure of the eukaryotic 80S ribosome in a translocation-intermediate state containing mRNA, the elongation factor eEF2 and tRNAs. The model demonstrates how the decoding centre releases a codon–anticodon duplex, allowing its movement on the ribosome. This experimental structure suggests how the 80S ribosome, eEF2 and tRNAs undergo large-scale molecular reorganizations to ensure maintenance of the mRNA reading frame during the complex process of translocation.

This article demonstrates Urania's scientists' capacity to solve high-resolution structures of eukaryotic ribosome complex, to understand at the molecular level the mechanism of the eukaryotic ribosome and to make use of this knowledge to design using a structure-based approach new drug candidates modulating the human ribosome.

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