Aptadir Therapeutics lands €40m seed led by 4BIO Capital to take its Fragile X RNA drug towards the clinic

Aptadir Therapeutics, a preclinical biotech based in Milan, has raised €40m (roughly $45m) in a seed round led by London life-sciences investor 4BIO Capital. The company calls it Italy's biggest biotech seed deal to date and among the largest in Europe, and it comes barely two years after Aptadir launched on a $1.6m pre-seed cheque.

The jump from $1.6m to €40m is the story for anyone who follows European biotech. Few Italian spinouts reach this kind of money before they have a drug in human trials, and Aptadir got there through a state-backed technology transfer programme rather than a classic venture studio.

A long list of backers

4BIO Capital led the round, which was announced on 29 September. EXTEND, Italy's national technology transfer hub for biopharma and Aptadir's first investor, came back for the seed. CDP Venture Capital joined through its Digital Transition Fund, alongside Indaco Venture Partners with two of its bio funds, XGEN Venture and CE-Ventures, the corporate venture arm of Crescent Enterprises.

Several investors also wrote cheques directly. According to Italian reports of the deal, they include Angelini Ventures, Kerna Ventures, Italian Angels for Biotech and Club degli Investitori. The mix of a foreign specialist lead, public capital, corporate funds and angel networks is typical of what a preclinical drug developer needs to reach the clinic, where costs climb fast.

Dima Kuzmin, managing partner at 4BIO, told Italian press the firm was drawn by how original the science is and by the chance to build more than one product on it. He said the conditions were in place, from the research to the management, to carry the technology into clinical development.

What the money pays for

Aptadir builds RNA medicines that work on DNA methylation, the chemical tagging that cells use to turn genes off. Its molecules, which it calls DNMT-interacting RNAs or DiRs, are designed to block faulty methylation at one chosen gene so that a gene wrongly shut down can work again. The company said it has shown this effect in disease models derived from patients.

The first target is Fragile X syndrome, an inherited condition that causes intellectual disability, learning problems and delays in development. The lead candidate is called CAP1-FMR1. The seed money is meant to move this and other programmes towards clinical development, and to fund further work in genetic diseases and in cancer.

That is a shift from the launch in 2024. Back then, the pre-seed was earmarked for refining a molecule called Ce-49, aimed at myelodysplastic syndromes, a group of blood cancers. Aptadir's own website still presents that programme as its lead, while the September announcement puts Fragile X first.

Giovanni Amabile, Aptadir's founder and chief executive, said the size of the round reflected both the DiRs platform and the international science team behind it. With the new funding, he said, the company can build a pipeline of candidates meant to restore gene pathways that "for decades, have been considered irreversibly silenced".

Four scientific founders and two CNR patents

The platform grew out of research by four scientific founders. Annalisa Di Ruscio and Daniel Tenen work at Beth Israel Deaconess Medical Center, Vittorio De Franciscis comes from Italy's National Research Council (CNR), and Marcin Kortylewski is at City of Hope National Medical Center. The Cancer Science Institute of Singapore was also involved in the underlying studies.

Di Ruscio's finding that certain RNAs bind to and inhibit the enzyme DNMT1 is the basis of the approach. In 2023 the company took an exclusive licence to two CNR patents covering this class of RNA inhibitors and their medical use. Amabile, who ran the Italian biotech Enthera and was earlier chief scientific officer at ADIENNE Pharma, joined as executive chairman and acting CEO at launch.

EXTEND itself was set up in 2022 by CDP Venture Capital together with Evotec and Angelini Ventures. Its job is to take research from labs, build a company around it and keep it alive until specialist investors will fund the next stage. Aptadir was the first company it created.

Claudia Pingue, who heads CDP Venture Capital's technology transfer fund and chairs EXTEND, said the hub saw early how strong Aptadir's science was. That conviction, she said, is why it helped form the company and backed it at the riskiest point, first in the pre-seed and now again in the seed.

For Italy, where research-born startups often run short of capital before their technology can be proven, the deal shows one path from patent to a large international round. CDP's technology transfer fund, worth €380m, is built around this kind of relay between public hubs and private investors.

None of this removes the scientific risk. Aptadir is still preclinical, and the €40m has to pay for the experiments that will show whether CAP1-FMR1 and the programmes behind it can work in people. The next milestone to watch is the step into human trials for its Fragile X candidate.