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§ Private Profile · Via San Marco 21A, 20121 Milano (MI), Italy
Biotech company developing RNA inhibitor therapeutics for oncology and genetic disorders, focused on gene reprogramming.
Aptadir Therapeutics is a Milan, Italy-based biotechnology company developing RNA inhibitor-based therapeutics to treat intractable cancers and rare genetic conditions by blocking aberrant DNA methylation and reprogramming genes. The organization's platform generates DNMTs Interacting RNAs for multiple suppressed genes, with its lead program, Aptadir Ce-49, utilizing nanoparticles for myeloid-specific delivery to target Myelodysplastic Neoplasms and leukemia. Operating as a pre-seed stage enterprise under the leadership of acting chief executive officer and executive chairman Giovanni Amabile, the company secured $1.6 million in initial funding in September 2024. This capital will advance their preclinical assets toward initial human clinical trials projected for late 2025, backed by institutional investors including CDP Venture Capital SGR, Angelini Ventures, and Evotec. Aptadir Therapeutics was founded in 2024 by Annalisa Di Ruscio, Vittorio De Franciscis, Daniel G. Tenen, and Marcin Kortylewski.
Aptadir Therapeutics has raised $2.0M across 1 funding round.
Aptadir Therapeutics has raised $2.0M in total across 1 funding round.
Aptadir Therapeutics has raised $2.0M across 1 funding round. Most recently, it raised $2.0M Seed in September 2024.
| Date | Round | Lead Investors | Other Investors | Status |
|---|---|---|---|---|
| Sep 1, 2024 | $2M Seed | CDP Venture Capital | — | Announced |
Aptadir Therapeutics has raised $2.0M in total across 1 funding round.
Aptadir Therapeutics's investors include CDP Venture Capital.
Aptadir Therapeutics is a newly launched biotech startup developing RNA-based therapeutics called DNMTs Interacting RNAs (DiRs) to reactivate silenced genes in intractable cancers and genetic disorders by targeting aberrant DNA methylation.[1][2][3] Its lead candidate, Aptadir Ce-49, addresses Myelodysplastic Syndromes (MDS), a blood cancer linked to hypermethylation, with plans to optimize preclinical development and enter clinical trials by the end of 2025; the platform also shows potential for rare diseases like Fragile X Syndrome.[1][3][4][5] Aptadir serves patients with oncology indications and genetic conditions where gene silencing hinders treatment, solving the core problem of epigenetic gene suppression without altering DNA sequences, offering selective, stable, and non-toxic reactivation at the single-gene level.[1][2][3]
Aptadir Therapeutics launched in September 2024, founded by leaders in RNA biology and epigenetics: Annalisa Di Ruscio, MD, PhD (Beth Israel Deaconess Medical Center, Harvard Medical School), Vittorio De Franciscis, PhD (Italian National Research Council), Daniel G. Tenen, MD (expert in MDS and leukemia, Cancer Science Institute of Singapore), and Marcin Kortylewski, PhD (expert in oligonucleotide immunotherapeutics, City of Hope National Medical Center).[2][3][4] The idea emerged from their landmark discovery of DiRs, a novel class of RNA molecules that inhibit DNA methyltransferases to reverse gene silencing—a key mechanism in cancers and genetic diseases.[1][3] Giovanni Amabile, PhD, was appointed executive chairman and acting CEO at launch, with board members Simone Braggio and Davide Santi; early focus centers on preclinical advancement of Aptadir Ce-49 for MDS, backed by initial funding.[1][2][3]
Aptadir rides the wave of precision epigenetics, targeting DNA methylation—a driver in 20-30% of cancers and disorders like Fragile X—where traditional gene therapies fall short due to off-target effects.[1][3] Timing aligns with surging investment in RNA therapeutics post-mRNA vaccine success, combined with advances in single-gene editing tools, amplifying DiRs' potential to reverse disease at the root.[1][4] Favorable market forces include unmet needs in MDS (poor prognosis, limited therapies) and rare diseases, plus growing biotech focus on adult-onset genetic fixes and pediatric syndromes.[2][3] Aptadir influences the ecosystem by validating RNA inhibitors for epigenetics, potentially inspiring platforms for hypermethylation-driven conditions and accelerating preclinical-to-clinic transitions in RNA biotech.[1][5]
Aptadir's near-term priority is advancing Aptadir Ce-49 through preclinical optimization into 2025 clinical trials for MDS, with parallel exploration of DiRs for Fragile X and other indications.[1][3][4] Rising trends in epigenetic reprogramming and RNA delivery tech (e.g., improved stability) will propel its platform, while partnerships with academic powerhouses like Harvard and CNR could de-risk development.[2][3] Influence may evolve from early-stage innovator to leader in gene reactivation if clinical data validate DiRs' selectivity, unlocking a pipeline for oncology and rare diseases—transforming Aptadir from stealth biotech to ecosystem shaper in precision medicine.[1][5] This positions it squarely at the intersection of its founding breakthrough and unmet patient needs.