# AptaDir Therapeutics

Source: https://onco.cc/companies/aptadir-therapeutics/  
OnCo record `aptadir-therapeutics` (Company). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

AptaDir Therapeutics is developing short RNA medicines that switch back on genes a cancer has silenced, by blocking the enzyme that adds the chemical tags which turn those genes off. Its first candidate is for myelodysplastic syndromes, a group of bone marrow cancers, and was preclinical in 2024.

## Summary

AptaDir Therapeutics launched in Milan on 24 September 2024 as the first company backed by EXTEND, the Italian national technology transfer hub set up by CDP Venture Capital with Angelini Ventures and Evotec, with USD 1.6 million in pre-seed funding. Its founders are Annalisa Di Ruscio (Beth Israel Deaconess Medical Center, Harvard Medical School), Vittorio De Franciscis (Italian National Research Council), Daniel Tenen and Marcin Kortylewski, and Giovanni Amabile was appointed executive chairman and acting chief executive.

The company is built on DNMT1-interacting RNAs (DiRs), first described by Di Ruscio and colleagues in Nature in 2013, which bind and inhibit DNA methyltransferase 1 and can block aberrant DNA methylation at a single gene. Its platform includes modified short RNA oligonucleotides for global DNMT1 inhibition (aptaDiR) and gene-specific fusions (captaDiR). The lead programme, Ce-49, is in development for myelodysplastic syndromes, with non-small cell lung cancer, Fragile X syndrome and metabolic disorders also listed in its pipeline. At launch the company aimed to enter the clinic by the end of 2025.

## Fields

- Kind: Company
- Last checked: 2026-10-29
- Also known as: Aptadir
- HQ: Milan
- Type: biotech
- Website: https://aptadir.com/

## Notes

- Founding year not stated. Whether Ce-49 entered the clinic by the end of 2025, as planned at launch, was not confirmed. The September 2026 seed amount comes from the ASGCT landscape report only.

## Sources

- Official website: https://aptadir.com/
- Launch press release, September 2024 (PDF): https://aptadir.com/wp-content/uploads/2024/09/Aptadir-Press-Release-24092024.pdf
- Pipeline: https://aptadir.com/projects/

## Connected records

- cancers: [Myelodysplastic syndromes / neoplasms (MDS)](https://onco.cc/cancers/mds/), [Non-small-cell lung cancer](https://onco.cc/cancers/nsclc/)
- fronts: [Epigenetic & Transcriptional Therapy](https://onco.cc/fronts/epigenetics/)
- technologies: [Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)](https://onco.cc/technologies/epigenetic-drugs/)

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