Deep Genomics claims AI drug discovery world first

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AI therapeutics firm Deep Genomics is claiming a world first after using artificial intelligence to identify a therapeutic drug candidate.

While many companies are using AI as a guide in the process, the Canadian firm’s CEO Brendan Frey said at the Elevate Festival in Toronto that this is the first time an AI platform has identified and confirmed multiple genetic variants that cause Wilson’s disease, and found a drug candidate that matches the target.

Last month Hong Kong’s Insilico Medicine said it found a way to use AI and deep learning to design, synthesise and validate a novel drug candidate in 46 days – 15 times faster than the best pharma companies.

While Insilico’s system used AI for virtual screening, Deep Genomics did all the work, including suggesting the disease target.

Wilson disease is a rare condition that causes excess copper to build up in organs, and has been targeted by rare diseases pharma companies such as Alexion.

Deep Genomics’ work has led to the identification of a drug candidate for the potentially life-threatening genetic condition.

The company will develop the candidate, DG12P1, for the treatment of  patients with the disease, who harbour a genetic mutation that impairs the body’s ability to remove copper.

Deep Genomics’ AI system scanned over 2,400 diseases and over 100,000 pathogenic mutations while searching for good drug development opportunities.

Analysing hundreds of millions of data points, the AI platform was able to predict and confirm the precise disease-causing mechanism of the Met645Arg mutations.

This is one of several genetic mutations leading to loss of function of the ATP7B copper-binding protein – a clear therapeutic target.

AI was then able to identify 12 lead candidates from thousands of potential compounds, taking into account in vitro efficacy and toxicity.

DG12P1 has been designed to correct the exon skipping mechanism of Met645Arg and, after tolerability experiments, Deep Genomics selected it for clinical development.

Wilson disease affects approximately one in every 30,000 people worldwide and, if left untreated, can cause life-threatening organ damage.

Brendan Frey, founder and CEO of Deep Genomics, said: “This is an important milestone for patients affected by Wilson disease and it represents a significant advance in the drug discovery community more broadly.

“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.

“We are delighted to nominate the first ever AI-discovered therapeutic candidate and are eager to move it rapidly into the clinic for the potential benefit of patients with Wilson disease and it represents a significant advance in the drug discovery community more broadly.”