Aptadir Therapeutics (Aptadir), a biotech company developing a novel class of RNA inhibitor-based therapeutics for treating intractable genetic conditions, announces the closing of one of the largest ever European seed rounds at €40m in life sciences and the largest round in Italy.

The lead investor is 4BIO Capital. Follow-on investment came from the original pre-seed investors, the EXTEND which is the Italian National Technology Transfer Hub initiated by CDP Venture Capital SGR and joint funded by Angelini Ventures and Evotec SE. New investors include CDP Venture Capital (through the Digital Transition Fund, which uses resources from the NextGenerationEU initiative to support the digital transition of supply chains and small and medium-sized enterprises), Xgen Ventures, Indaco Bio, through the Indaco Bio Fund and Bio Lombardia FundCE-Ventures, funded with the support of the European Regional Development Fund (ERDF) under the 2021–2027 Regional Programme of the Lombardy Region, Angelini Ventures through direct investment, Kerna Ventures, Italian Angels for Biotech and the the members of Club degli Investitori, through Simon Fiduciaria S.p.A.

The funds will be used to grow the Company and advance its pipeline of disease-modifying investigational RNA therapeutics, including its lead candidate, CAP1-FMR1 for Fragile X Syndrome, towards clinical development. The Company’s technology is based on the landmark discovery of a new class of RNAs which are capable of blocking aberrant DNA methylation at a single gene level1,2. By reversing this abnormal gene silencing, Aptadir’s RNA inhibitors can reactivate gene expression and restore normal biological function, an effect the Company has demonstrated in complex patient-derived disease models. Aptadir is developing a portfolio of drug candidates based on this proprietary novel RNA inhibitors technology. The molecules are called DNMTs Interacting RNAs (DiRs) which target specific genes that are suppressed in cancer and genetic conditions. The technology is based on the findings of an international group of renowned scientists in the fields of RNA and DNA biology, genetic disorders, Leukemia and immunotherapeutics including Annalisa Di Ruscio and Daniel Tenen from the Beth Israel Deaconess Medical Center, Vittorio de Franciscis of the Italian Research National Council, Marcin Kortylewsky of City of Hope National Medical Center. 

Giovanni Amabile, CEO, said: “The magnitude of this seed round reflects of the exciting potential of our DiRs technology and the quality of truly international scientific team. Originating from world-class institutions, the discovery of this new class of RNA inhibitors was a landmark event not only from a scientific point of view, but also for providing new found hope for patients affected by intractable disorders. With the backing of our investors, we now have the opportunity to develop a pipeline of drug candidates with the potential to restore genetic pathways that, for decades, have been considered irreversibly silenced.”

Dima Kuzmin, Managing Partner of lead investor, 4BIO Capital, added: “Finding a truly innovative new science with such potential is a rare event, so we are delighted to be leading one of the largest seed rounds in Europe and certainly the largest in Italy. The group of founders behind the science are world-renowned and the potential of the technology could be life changing. We are confident that the right factors are in place, from the science to the management team, to take this technology forward into the clinic.”

Claudia Pingue, Head of Technology Transfer fund at CDP Venture Capital and Chairman of EXTEND said: “Our purpose at the EXTEND is to identify and help develop translational drug discovery opportunities. We recognized how powerful Aptadir’s science could be from the outset, and this was the rationale for us to spinning out the company and investing from the very earliest, highest risk stages, first at the pre-seed and follow through with this significant Seed round. We are proud that this science is now attracting further investment from international partners, and will continue to support the Company as it develops.”