Combining venture capital and capabilities in kind for drug development, this innovative operation will help fund the development of promising readthrough compounds for the advent of new drugs targeting monogenic diseases, a further step towards precision medicine

Strasbourg, France, November 6, 2019 – Urania Therapeutics, a biopharmaceutical company specializing in the identification and development of ‘readthrough’ compounds, today announces it has successfully raised €3.53M ($3.89M) in seed funding to unlock the therapeutic potential of ribosome-based treatments.

The seed round, led by Advent France Biotechnology, will allow Urania Therapeutics to consolidate its capabilities and optimize its proprietary compounds, enabling it to select drug candidates for further progress. This ambitious life science project combines venture capital from Advent France Biotechnology (AFB) and Cap Innov’Est (CIE) and strong capabilities, provided by NovAliX, a CRO specialized in medicinal chemistry and biophysics for drug development.

Urania Therapeutics is researching new therapeutic options for a wide range of monogenic diseases, by targeting the decoding centre of the human ribosome, thereby restoring production of functional protein. This will pave the way for new treatments for patients with rare monogenic diseases, such as Duchenne Muscular Dystrophy (DMD) and Cystic Fibrosis (CF), as well as certain cancer subtypes caused by nonsense mutations.

The readthrough approach has the potential to transform the current standard of care for orphan diseases, many of which have no treatments available. Since readthrough compounds target a gene-independent mechanism, this approach could lead to a common treatment for patients with these life-threatening conditions. According to analysts from Evaluate Pharma, orphan drugs represent a growing segment of the pharmaceutical market and are expected to almost double by 2024, reaching $239bn (€217bn).

Full press release

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