DG12P1

**DG12P1** is an antisense oligonucleotide therapy discovered by Deep Genomics using an AI-driven platform. It targets the **Met645Arg mutation (NM_000053.3:c.1934T>G)** in the **ATP7B** gene, which causes **exon 6 skipping** during RNA splicing, resulting in a frameshift, premature stop codon, and loss of functional ATP7B copper-transporting protein. This leads to copper accumulation in the liver and brain in **Wilson disease**, a rare genetic disorder affecting ~1 in 30,000 people. DG12P1 corrects the splicing defect to restore ATP7B function and enable proper copper excretion. Nominated in 2020 as the first AI-discovered therapeutic candidate after AI scanned 2,400+ diseases and 100,000+ mutations, evaluating thousands of compounds for efficacy and toxicity in vitro.[1][2][5][7][9]