Aptadir Therapeutics is pioneering a novel therapeutic approach that focuses on reprogramming DNA methylation through RNA therapeutics, specifically targeting the enzyme DNA methyltransferase 1 (DNMT1). Aberrant methylation caused by DNMT1 can lead to abnormal cell development and various diseases, including certain cancers and rare genetic disorders. The company has developed RNA-based platforms that leverage the discovery of DNMT1-interacting RNAs (DiRs), which bind to and inhibit DNMT1, resulting in locus-specific changes in DNA methylation and gene expression. This innovative approach allows for both global inhibition of DNMT1 using modified short RNA oligonucleotides (aptaDiR) and locus-specific inhibition through the fusion of aptaDiR with gene-specific sequences (captaDiR), providing a targeted and less toxic alternative to existing therapies.

The applications of Aptadir's technology span a wide range of diseases, including various cancers (such as lung, breast, and colon cancers), genetic disorders (like Fragile X syndrome and myotonic dystrophy), neurological conditions (including Alzheimer's disease), and autoimmune diseases (such as rheumatoid arthritis). By utilizing RNA-guided platforms, Aptadir aims to create selective and gene-specific DNMT inhibitors that can address the underlying causes of these disorders, offering potential new treatment options for conditions that are currently considered untreatable. The company's research is grounded in a strong scientific foundation, with multiple publications supporting the efficacy of their RNA-based approaches in modulating DNA methylation and gene expression.

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Reprogramming DNA methylation in disease conditions by RNA therapeutics APTADIR Contact us NEWS Press Release Reprogramming DNA methylation by RNA therapeutics A novel therapeutic approach for untreatable cancers and rare diseases Aberrant methylation by DNA methyltransferase 1 (DNMT1) of certain genes may cause abnormal cell development and lead to disease. Current therapies targeted to inhibit

Aptadir Therapeutics

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