Deep Genomics Advances What It Calls First AI Discovered Therapeutic Candidate

September 25, 2019

Deep Genomics said its proprietary artificial intelligence-based drug discovery platform has identified a novel treatment target and corresponding drug candidate for Wilson disease, a rare, serious, and potentially life-threatening genetic disorder.

Photo: Brendan Frey, founder and CEO of Deep Genomics

The company hailed it as the “first ever AI-discovered therapeutic candidate.”

Wilson disease can be caused by several different mutations that lead to loss of a protein required for copper transport (ATP7B). Without the necessary protein, copper is improperly regulated in the body and accumulates at toxic levels in the liver and central nervous system, leading to hepatic, neurological and psychiatric symptoms. The standard of care presents serious adverse effects and adherence issues. Wilson disease affects approximately one in every 30,000 people worldwide and, if left untreated, can cause life-threatening organ damage.

Deep Genomics plans to develop its experimental therapy, DG12P1, for the treatment of patients with Wilson disease who have a genetic mutation that impairs the body’s ability to remove copper.

“This is an important milestone for patients affected by Wilson disease, and it represents a significant advance in the drug discovery community more broadly,” said Brendan Frey, founder and CEO of Deep Genomics. “Within 18 months of initiating our target discovery effort, we identified a genetic mutation that causes the disease, the chemical properties needed in a molecule to target the mutation, and a compound that warrants further investigation.”

Deep Genomics’ AI system scanned more than 2,400 diseases and more than 100,000 pathogenic mutations while searching for drug development opportunities. By analyzing hundreds of millions of data points, Deep Genomics said its AI platform was able to predict and confirm the precise disease-causing mechanism of the mutation Met645Arg, one of several genetic mutations that leads to loss of function of the ATP7B copper-binding protein, and thereby identify a clear therapeutic target.

The AI system was then used to identify 12 lead candidates out of thousands of potential compounds, taking into account in vitro efficacy and toxicity. DG12P1 was designed to correct the exon skipping mechanism of Met645Arg and, after tolerability experiments, Deep Genomics declared it the ideal candidate to advance toward IND.

In a subsequent analysis, Deep Genomics’ AI platform was able to rapidly identify two additional mutations that can cause Wilson disease. These potential therapeutic targets are currently being experimentally confirmed.

“Our expectation is that, going forward, Deep Genomics’ platform will enable them to go from known target to first patient dosed in less than half the time of the industry standard, and they may be able to do this even faster with subsequent programs,” said Arthur Levin, a member of the company’s strategic advisory board. “This is truly unprecedented and opens the door to a smarter, faster, and vastly more efficient means of identifying viable drug candidates for a host of diseases. Developing new therapeutics is full of unknowns, but I am certain that we are witnessing a new era of drug discovery.”

Author: Rare Daily Staff

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