In the ever-evolving landscape of medical science, a new frontier is being charted by an Italian startup, which is pioneering a novel class of RNA-based drugs.
These innovative therapies are poised to revolutionize the treatment of intractable cancers and genetic disorders, offering new hope where traditional methods have fallen short.
The potential of RNA-based therapies to transform healthcare is immense, particularly in oncology, rare diseases, infectious diseases, and neurodegenerative disorders.
These treatments leverage ribonucleic acid (RNA) to alter gene expression or protein production, a method that is gaining significant traction among biopharmaceutical and biotechnology companies.
The growing demand for personalized medicine and the ability of these therapies to address unmet medical needs are driving substantial investments in this field.
According to Technavio, the global market for RNA-based therapies is projected to expand by $1.26 billion by 2028.
Various classes of RNA therapies are under development, including messenger RNA, small interfering RNA, and aptamers.
Adding to this burgeoning field is a new category developed through Italian research, leading to the creation of Aptadir Therapeutics.
This biotech company specializes in therapies based on novel RNA inhibitors known as DNMT interacting RNA (Dir), aimed at treating intractable cancers and genetic disorders.
The launch of Aptadir, the first investment by the joint venture Extend, was announced by Giovanni Amabile, the executive chairman and interim CEO. Amabile explains that they have transformed a specific class of RNA into aptamers, potential drugs, based on the discovery by Italian hematologist Annalisa di Ruscio, an assistant professor at Harvard Medical School and co-founder of Aptadir with Vittorio De Franciscis from CNR. Their research, published in 2023 in Nature Communication, revealed a cellular mechanism that defends against cancer development by blocking the methylation of tumor suppressor genes.
When these genes are methylated, cellular proliferation goes unchecked, leading to cancer progression.
This discovery led to the development of a drug targeting myelodysplastic syndrome, a condition with a high risk of acute myeloid leukemia.
The same mechanism is also present in some rare genetic disorders, such as fragile X syndrome, which currently lacks treatment.
Aptadir has developed another molecule targeting this specific gene, acting as a shield against methylating proteins.
The primary challenge for biotech companies in this field is to specifically target the tissue involved in the pathological process.
Aptadir is addressing this by engineering lipid nanoparticles to recognize the surface of target cells, advancing two research lines: one focused on methylation inhibitors and the other on creating a delivery vehicle for the drug.
Amabile notes that RNA technology could gradually replace gene therapy, as it is safer without the need for viral vectors, and RNA therapeutics are more cost-effective and faster to produce industrially.
Aptadir has secured $1.6 million in pre-seed funding from the Extend joint venture to optimize their first molecule, Aptadir Ce-49, before clinical trials.
Amabile concludes that the Dir inhibitors are highly selective, stable, non-toxic, and have the potential for various therapeutic applications.
The company aims to raise 10-15 million euros to advance the drug into clinical development.

Insights

RNA-based therapies represent a significant shift in medical treatment paradigms, offering targeted and personalized approaches to complex diseases.

The ability to modulate gene expression and protein production opens new avenues for treating conditions previously deemed untreatable.
The development of RNA therapeutics is a testament to the power of interdisciplinary collaboration, combining insights from molecular biology, genetics, and pharmacology.

Opportunities

The burgeoning field of RNA-based therapies presents numerous opportunities:

1. Development of personalized medicine tailored to individual genetic profiles. 2. Expansion into new therapeutic areas, including infectious and neurodegenerative diseases. 3. Collaboration between biotech companies and academic institutions to accelerate research and development. 4. Investment opportunities in emerging biotech firms specializing in RNA therapeutics. 5. Advancements in delivery systems, such as lipid nanoparticles, to enhance the efficacy and specificity of RNA-based treatments.