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Insilico Medicine Identifies multiple new targets for ALS

July 8, 2022

AI drug discovery company Insilico Medicine said it has identified multiple, unreported potential therapeutic targets for the rare, neurodegenerative condition amyotrophic lateral sclerosis using its PandaOmics discovery engine.

The research, performed in collaboration with Answer ALS, was published in the June 28 issue of Frontiers in Aging Neuroscience.

Globally, more than 700,000 people live with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. People with ALS lose voluntary muscle movement and therefore the ability to walk, talk, eat and, eventually, breathe. Progression of ALS disease is generally rapid, with patients facing an average life expectancy of between two and five years from the onset of symptoms. Unfortunately, existing approved drugs for ALS do not halt or reverse the loss of function.

The team of researchers leveraged massive datasets to find genes relevant to disease, which could serve as potential targets for new therapeutics. PandaOmics, Insilico’s proprietary AI-driven target discovery engine, helped analyze the expression profiles of central nervous system (CNS) samples from public datasets, and direct iPSC-derived (diMN) motor neurons from Answer ALS.

As a result of the study, 17 high-confidence and 11 novel therapeutic targets were identified from CNS and diMN samples. These targets were further validated in c9ALS Drosophila model, mimicking the most common genetic cause of ALS, of which 18 targets (64 percent) have been validated to have functional correlations to ALS. Notably, eight unreported genes, including KCNB2, KCNS3, ADRA2B, NR3C1, P2RY14, PPP3CB, PTPRC, and RARA, rescue neurodegeneration through their suppression strongly. All the potential therapeutic targets were disclosed in the paper and at ALS.AI.

“The results of this collaborative research effort show what is possible when we bring together human expertise with AI tools to discover new targets for diseases where there is a high unmet need,” said Alex Zhavoronkov, founder and CEO of Insilico Medicine. “This is only the beginning.”

Photo: Alex Zhavoronkov, founder and CEO of Insilico Medicine

Author: Rare Daily Staff

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