Aptadir Therapeutics is developing RNA inhibitor-based therapeutics that selectively inhibit DNA methyltransferase 1 (DNMT1) to reprogram aberrant DNA methylation associated with intractable cancers and genetic conditions. Their platform utilizes modified RNA oligonucleotides for global inhibition and gene-specific sequences for locus-specific modulation, offering a targeted approach with reduced toxicity compared to existing therapies.
Funding
$1.6M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
ESFounders
Product
Problem
Aberrant DNA methylation, often caused by DNA methyltransferase 1 (DNMT1), can lead to abnormal cell development and contribute to intractable cancers and genetic conditions. Current therapies targeting DNMT1 lack gene specificity, exhibit short-term results, and induce significant toxicity.
Solution
Aptadir Therapeutics is developing RNA-based therapeutics that selectively inhibit DNMT1 to reprogram aberrant DNA methylation associated with disease. Their platform utilizes modified RNA oligonucleotides to achieve both global and locus-specific DNMT1 inhibition. For global inhibition, short RNA oligonucleotides (aptaDiR) are used to potently sequester DNMT1 without causing cell toxicity. For locus-specific inhibition, the aptaDiR is fused with a gene-specific sequence (captaDiR) to enable targeted modulation of DNA methylation at specific genomic locations. This approach offers a more targeted therapy with potentially reduced toxicity compared to existing DNMT1 inhibitors.
Target Audience
The primary target audience includes patients with intractable cancers and genetic conditions characterized by aberrant DNA methylation, as well as researchers and clinicians focused on epigenetic therapies.
Features
- Modified short RNA oligonucleotides (aptaDiR) for global DNMT1 inhibition
- Gene-specific sequence-fused aptaDiR (captaDiR) for locus-specific DNA methylation modulation
- Selective DNMT1 inhibition to minimize off-target effects
- Reduced toxicity compared to existing DNMT1-targeting therapies