Deep Genomics Identifies AI-Discovered Candidate for Wilson Disease

GEN | September 25, 2019

A Toronto developer of treatments based on artificial intelligence said today it has successfully used its own AI drug discovery platform to identify its first therapeutic candidate, a therapy for Wilson disease that the company plans to advance into clinical studies in 2021. Deep Genomics says that its initial candidate, DG12P1, is an oligonucleotide therapy designed to treat Wilson disease in patients who possess the mutation of Met645Arg—a mutation shown to lead to the loss of function of the ATP7B copper-binding protein. The mutation is among an unknown number of mutations linked to Wilson disease, which causes copper to accumulate in the liver, brain, and other vital organs. The rare and potentially life-threatening inherited disorder affects approximately one in every 30,000 people worldwide, as noted in a 2015 review article. In a study preprint published September 17 in bioRxiv, Deep Genomics founder and CEO Brendan Frey, PhD, FRSC, and colleagues, reported that they used a minigene system and gene-edited HepG2 cells to show that the variant—also known as NM_000053.3:c.1934T>G, and highly prevalent among Wilson Disease patients of Spanish descent—caused approximately 70% skipping of exon 6. That exon skipping, in turn, resulted in frameshift and stop gain, which is expected to cause loss of ATP7B function.

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Omega Therapeutics, Inc | October 14, 2021

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Acticor Biotech | October 05, 2020

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CELL AND GENE THERAPY

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2seventy bio, Inc | January 07, 2022

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