04 ott 2024
An Italian startup is pioneering a new class of RNA-based drugs aimed at treating intractable cancers and genetic disorders.
Aptadir Therapeutics, a biotech company, is developing RNA inhibitors known as Dnmt interacting RNA (Dir) to target gene methylation processes that contribute to cancer progression.
This approach, which could potentially replace gene therapy, is seen as safer and more cost-effective.
The company has secured initial funding to optimize its first molecule, with plans to enter clinical trials and expand its therapeutic applications.
A groundbreaking development in the field of RNA-based therapies has emerged from an Italian startup, Aptadir Therapeutics.
This biotech company is focusing on creating a new class of RNA inhibitors, known as Dnmt interacting RNA (Dir), to address the challenges of treating intractable cancers and genetic disorders.
RNA-based therapies, which utilize ribonucleic acid to alter gene expression or protein production, hold significant promise in revolutionizing healthcare, particularly in oncology, rare diseases, infectious diseases, and neurodegenerative disorders.
The global market for RNA-based therapies is projected to grow substantially, driven by the demand for personalized medicine and the potential to address unmet medical needs.
Aptadir's innovative approach involves transforming specific RNA classes into potential drugs, a concept initially discovered by hematologist Annalisa di Ruscio.
Her research revealed a cellular mechanism that defends against cancer by blocking the methylation of tumor suppressor genes.
This discovery has led to the development of a drug targeting myelodysplastic syndrome, a condition with a high risk of acute myeloid leukemia.
Additionally, the same mechanism is applicable to certain rare genetic disorders, such as fragile X syndrome, which currently lacks effective treatment.
The challenge for biotech companies in this field is to specifically target the affected tissue.
Aptadir is addressing this by engineering lipid nanoparticles to recognize target cell surfaces, advancing two research lines: one focused on methylation inhibition and the other on drug delivery vehicles.
RNA technology offers a safer alternative to gene therapy, as it does not require viral vectors, and is more cost-effective and faster to produce.
Aptadir has received $1.6 million in pre-seed funding from the joint venture Extend to optimize its first molecule, Aptadir Ce-49, before clinical trials.
The Dir inhibitors have shown high selectivity, stability, and non-toxicity, with potential applications in various therapeutic areas.
The company aims to raise an additional €10-15 million to advance the drug into clinical development.