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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.
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.
Investors not recorded; see the round sources.
| Round | Year | Amount | Source |
|---|---|---|---|
| Seed · EUR 40 million seed listed for 29 September 2026 in the ASGCT and Citeline Q3 2026 landscape report; the company's own release was not read | 2026 | undisclosed | asgct.org |
| Pre-seed · From EXTEND, the Italian national technology transfer hub set up by CDP Venture Capital with Angelini Ventures and Evotec | 2024 | $2M | aptadir.com |
Amounts only where the cited source states them. Rounds without a public source are not listed.
Shares Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Myelodysplastic syndromes / neoplasms (MDS).
Shares Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Non-small-cell lung cancer.
Shares Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Non-small-cell lung cancer.
Shares Myelodysplastic syndromes / neoplasms (MDS), Non-small-cell lung cancer.
Shares Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Myelodysplastic syndromes / neoplasms (MDS).
Shares Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Non-small-cell lung cancer.
Shares Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Myelodysplastic syndromes / neoplasms (MDS).
Shares Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET), Myelodysplastic syndromes / neoplasms (MDS).
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