Italian Startup Raises $1.6M to Combat Cancer with RNA Inhibitors
Milan, Friday, 27 September 2024.
Aptadir Therapeutics launches with $1.6 million to develop innovative RNA inhibitors targeting DNA hypermethylation in cancer and genetic disorders. The Milan-based startup aims to begin clinical trials by early 2026, focusing on myelodysplastic syndrome and Fragile X syndrome.
Understanding RNA Inhibitors
RNA inhibitors are an emerging class of therapeutic agents designed to target and modulate the activity of specific RNA molecules within cells. Aptadir Therapeutics is leveraging this innovative technology to address diseases caused by DNA hypermethylation, a process that can lead to the silencing of critical genes involved in cell differentiation and growth. Hypermethylation often affects genes such as onco-suppressors in cancer and the FMR1 gene in Fragile X syndrome (FXS), which impacts approximately 1 in 11,000 females and 1 in 7,000 males[1].
The Science Behind Aptadir’s Approach
Aptadir’s approach involves transforming non-coding RNA into aptamers, which are short, single-stranded RNA molecules that can bind to specific targets with high affinity. These aptamers act as a shield against hypermethylation by blocking the DNA methyltransferase 1 (DNMT1) enzyme from attaching to the DNA. This not only prevents additional methylation but also progressively reactivates the gene, as demonstrated in their preclinical studies. Giovanni Amabile, the executive chairman of Aptadir, explained that their technology has shown promising results in both cancer cell lines and cells affected by Fragile X syndrome[1].
Clinical Prospects and Challenges
Aptadir plans to initiate clinical trials for their RNA inhibitors targeting myelodysplastic syndrome (MDS) and chronic myeloid leukemia by late 2025 or early 2026. Additionally, they are developing treatments for Fragile X syndrome using induced pluripotent stem cells. One of the key challenges highlighted by Amabile is the toxicity of current compounds, which many patients find intolerable. By focusing on early intervention, particularly in pediatric cases of Fragile X syndrome, Aptadir aims to maximize the efficacy and minimize the adverse effects of their treatments[1].
Market and Future Directions
The successful launch of Aptadir Therapeutics, with a $1.6 million pre-seed funding round completed on 24 September 2024, underscores the growing interest in RNA-based therapies within the biotech industry[2]. As the company prepares for clinical trials, it is also working on identifying potential biomarkers and conducting manufacturing risk assessments to scale up its lead assets. The innovative use of RNA inhibitors could revolutionize the treatment landscape for diseases caused by DNA hypermethylation, offering new hope for patients with conditions that currently have limited therapeutic options[1].