Aptadir Therapeutics, a biotech company developing a new class of RNA inhibitor-based therapies for the treatment of intractable genetic disorders, has announced that it has closed a seed funding round worth 40 million euros.
The funding round is led by 4BIO Capital, with participation from pre-seed investor Extend, the Italian National Technology Transfer Centre promoted by CDP Venture Capital and co-financed by Angelini Ventures and Evotec SE.
Other participants in the seed round included CDP Venture Capital, through the Digital Transition Fund, Indaco Venture Partners (through Indaco Bio Structure and Indaco Bio Parallel Lombardia), XGEN Venture and CE-Ventures. Other participants included Angelini Ventures, with a direct investment, Kerna Ventures, Italian Angels for Biotech and Club degli Investitori.
The funds will be allocated to advancing the pipeline of experimental RNA therapies with disease-modifying potential, starting with the lead candidate, CAP1-FMR1, for the treatment of Fragile X syndrome. The company’s technology is based on the fundamental discovery of a new class of RNA capable of blocking aberrant DNA methylation at the single-gene level (Di Ruscio A et al 2013, Nature and Esposito CL et al 2023, Nature Communications). By reversing this abnormal gene silencing, Aptadir’s RNA-based inhibitors are able to reactivate gene expression and restore normal biological function, an effect that the company has demonstrated in complex disease models derived from patient tissues.
Aptadir is developing a portfolio of experimental drugs based on this proprietary technology. The molecules, known as DNMTs Interacting RNAs (DiRs), act on specific genes that are silenced in tumours and genetic diseases.
The Aptadir platform stems from research carried out by a group of internationally renowned scientists in the fields of RNA biology, genetic diseases, leukaemia and immunotherapy, including Annalisa Di Ruscio and Daniel Tenen of the Beth Israel Deaconess Medical Centre, Vittorio de Franciscis of the National Research Council and Marcin Kortylewsky of the City of Hope National Medical Centre.
Giovanni Amabile, founder and CEO (pictured), said in a statement: “The size of this seed round reflects the great potential of our DiRs technology and the calibre of our truly international scientific team. Originating in world-class institutions, the discovery of this new class of RNA inhibitors has represented a breakthrough not only from a scientific perspective, but also because it offers new hope to patients suffering from conditions that have for years been considered incurable. With the support of our investors, we now have the opportunity to develop a pipeline of experimental drugs capable of restoring the function of specific genes that for decades have been considered irreversibly silenced.”
Dima Kuzmin, managing partner of the lead investor, 4BIO Capital, adds: “Finding a truly innovative science with such potential is a rare occurrence. We are delighted to support this new therapeutic approach, which could give rise to a range of treatments targeting disease mechanisms that have not yet been fully addressed by existing drugs. The founding team behind this science is world-renowned, and the technology developed by the company has the potential to change patients’ lives. We are convinced that all the right factors are in place – from the science to the management team – to take this platform through to the clinical phase.”
Claudia Pingue, head of the Technology Transfer Fund at CDP Venture Capital and chair of Extend, says: “Extend’s aim is to identify opportunities in translational drug discovery and contribute to their development. We recognised the strength of Aptadir’s scientific research right from the start: that is why we chose to set up the company as a spin-off and to invest from the very earliest, highest-risk stages – first with pre-seed funding and now with this significant seed round. We are proud that this line of research is attracting further investment from international partners and we will continue to support the company on its growth journey.”
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