Aptadir secures €40M for gene-reactivating RNA drugs
Aptadir Therapeutics has raised €40 million in seed funding to advance an RNA therapy designed to reactivate the gene silenced in Fragile X syndrome.
Why it matters: The Milan-based company is trying to reverse the gene silencing that causes the condition, rather than treat its downstream effects. Aptadir says that its lead candidate, CAP1-FMR1, has restored gene expression and function in patient-derived disease models.
How it works: Fragile X syndrome is caused by silencing of the FMR1 gene, which sharply reduces production of FMRP, a protein needed for normal brain development. Aptadir is developping what the company calls DNMT-interacting RNAs (DiRs) designed to interfere with DNMT1, an enzyme that helps maintain chemical marks on DNA that can silence genes. Aptadir aims to use them to reactivate FMR1 and restore the production of FMRP. The underlying work was described in two Nature papers published in 2013 and 2023.
Zoom in: 4BIO Capital led the round. Returning investor EXTEND participated alongside CDP Venture Capital, Indaco Venture Partners, XGEN Venture, CE-Ventures, Angelini Ventures, Kerna Ventures, Italian Angels for Biotech and Club degli Investitori. Aptadir says the money will support CAP1-FMR1 and other programs built on its RNA platform.
Backstory: Aptadir launched in 2024 with $1.6 million in pre-seed funding from EXTEND, an Italian technology transfer initiative backed by CDP Venture Capital, Angelini Ventures and Evotec. At the time, its first program was Ce-49 for myelodysplastic syndrome, a group of blood cancers. The company aimed to take that candidate into the clinic by the end of 2025 but doesn’t seem to have achieved the intended milestone.
What to watch: Fragile X is now the lead program in Aptadir’s announcement. The company has not given a timeline for taking CAP1-FMR1 into human trials, but the sizable seed round will certainly help to accelerate the development of the drug candidate.
