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Aptadir Therapeutics Launches With $1.6M to Advance RNA Inhibitors for Cancers, Genetic Conditions

NEW YORK – Aptadir Therapeutics launched on Tuesday with the goal of advancing its RNA inhibitor-based drug candidates for cancers and genetic conditions, beginning with Aptadir Ce-49, its lead candidate for myelodysplastic syndrome (MDS). 

The Milan-based firm is developing what it calls DiRs, or DNA methyltransferases (DNMTs) Interacting RNAs. The RNA inhibitors work by blocking aberrant DNA methylation at a single-gene level, in turn reactivating genes that were previously hypermethylated and contributing to cancer or genetic disorders such as MDS or fragile X syndrome. For MDS, Aptadir is targeting DNA methyltransferase 1 (DNMT1), and for fragile X syndrome, the firm is targeting FMR1, the gene that causes the disease when mutated. 

"MDS is a condition in which several genes become progressively hypermethylated, including several onco-suppressors such as RASSF1, HIC1, and FHIT, [and] for this reason, it is not possible to target a specific gene," Aptadir CEO and Executive Chairman Giovanni Amabile told Precision Medicine Online. "Thus our molecule directly targets DNMT1." 

The product, Aptadir Ce-49, has a delivery system that consists of nanoparticles that can target the myeloid compartment of a cell while sparing other tissues, he added. Aptadir anticipates starting to test Aptadir Ce-49 in clinical trials by the end of 2025. 

By reactivating specific genes, the firm hopes to target the underlying cause of these cancers and disorders, potentially reversing them. Aptadir developed these RNA inhibitors using a proprietary platform built to generate multiple DiRs for specific genes in cancer and genetic conditions. "From data already generated, DiRs are highly selective, stable, and nontoxic," Amabile said in a statement. "[They] have the potential to be used across multiple indications." 

The firm launched with $1.6 million in pre-seed funding from the EXTEND initiative, which it aims to use to advance Aptadir Ce-49. EXTEND is an initiative of the Italian National Technology Transfer Hub, which was started by CDP Venture Capital and jointly funded by Evotec and Angelini Ventures and is dedicated to supporting biopharma to develop new therapies. Aptadir is the first biotech launched under the EXTEND initiative.