A new generation of RNA based medicines is emerging around the idea that some diseases could be addressed not simply by treating their symptoms, but by changing the molecular processes that keep important genes switched off. Milan based biotech company AptaDir Therapeutics is pursuing that approach and has raised $45 million in seed funding to advance its pipeline of potential treatments for rare genetic diseases and difficult to treat cancers.
The round was led by London based life sciences investor 4BIO Capital, with participation from Extend, CDP Venture Capital, XGEN Venture and CE Ventures. The investment gives AptaDir resources to move its technology and drug candidates through the preclinical development process.
Targeting Gene Silencing
AptaDir’s platform is built around DNA methylation, a biological mechanism that can influence whether genes are active or silenced.
The company’s research focuses on DNA methyltransferase 1, or DNMT1, an enzyme involved in maintaining DNA methylation. According to AptaDir, abnormal methylation of certain genes can contribute to disease by preventing genes from being expressed normally.
Rather than using conventional small molecule drugs to inhibit DNMT1, AptaDir is developing RNA based approaches designed to interact directly with the enzyme.
Its technology originates from research showing that specific RNA molecules can bind to DNMT1 and prevent it from maintaining DNA methylation. This can potentially restore gene expression in selected biological pathways.
The DiR Technology
The company’s platform is based on two related approaches called aptaDiR and captaDiR.
AptaDiR uses modified short RNA molecules designed to sequester DNMT1 and inhibit its activity. AptaDir says this approach can achieve potent DNMT1 inhibition without the cellular toxicity associated with some existing approaches.
CaptaDiR takes the technology further by combining the aptaDiR component with a gene specific sequence. The objective is to create a more targeted mechanism capable of directing the RNA based inhibitor toward a particular genetic locus.
This distinction could become important for developing treatments where changing the activity of a specific gene or pathway is more desirable than broadly affecting DNA methylation.
Starting With Fragile X Syndrome
AptaDir’s initial development programme is focused on Fragile X Syndrome, a genetic condition associated with intellectual disability, learning difficulties and developmental challenges.
The company’s strategy is based on the possibility that certain disease related genes can be reactivated by modifying the molecular mechanisms responsible for their silencing. The technology is still at the preclinical stage, meaning its potential safety and effectiveness have yet to be established through human clinical trials.
The Fragile X programme represents the first application of a platform that AptaDir hopes can eventually be used across multiple genetic diseases and cancers.
A Broader Therapeutic Pipeline
Beyond rare genetic disorders, AptaDir is investigating applications in oncology and other diseases where abnormal DNA methylation plays an important role.
The company says its technology was developed from research involving scientists working across RNA and DNA biology, genetic disorders, leukaemia and immunotherapeutics. Its broader objective is to develop a pipeline of RNA therapeutics capable of addressing biological pathways that have historically been difficult to modify.
Scaling Italian Biotech
The $45 million financing provides AptaDir with significant capital for a company still operating at the preclinical stage. The funding will primarily support development of its therapeutic pipeline and advance its RNA based technology toward future clinical programmes.
AptaDir is headquartered in Milan, where the company continues to develop its research and therapeutic platform.
The funding also highlights growing investor interest in RNA therapeutics beyond established approaches such as vaccines and conventional gene silencing. For AptaDir, the goal is more specific: use RNA to influence the molecular machinery controlling gene expression and potentially restore pathways that are inactive in disease.
If its preclinical programmes successfully translate into human studies, the company could develop a new class of medicines aimed at genetic conditions and cancers where existing treatment options remain limited.
