Insilico Medicine Pinpoints New Targets for ALS With AI

After trawling through massive datasets with its AI discovery engine PandaOmics, New York-based Insilico Medicine and its collaborators have identified potential gene targets for amyotrophic lateral sclerosis, or ALS. Suppression of 18 out of the 28 identified genes may rescue neurodegeneration, follow-up preclinical research on fruit flies suggest.

The study is led by Insilico, with support from Answer ALS and researchers at Johns Hopkins University School of Medicine, Massachusetts General Hospital and Harvard Medical School, Mayo Clinic, University of Zurich, 4B Technologies, Limited, Tsinghua University, and the Buck Center for Aging Research.

The findings were published in the June 28 issue of Frontiers in Aging Neuroscience. 

Related article: Biogen’s ALS Drug Fails to Attain Primary Endpoint but Offers Hope With Secondary Outcomes

The AI Journey from Discovery to Cure 

PandaOmics analyzes trillions of data points from millions of samples, including microarrays, RNA sequences, and proteomes. It also goes over millions of documents, including patents, grants, publications, and clinical trial reports to identify brand new targets for drugs. 

In the study, PandaOmics analyzed expression profiles of central nervous system (CNS) from public datasets, and direct iPSC-derived motor neurons (DiMN) provided by Answer ALS.

From the samples, the AI-driven engine found 17 high confidence and 11 novel therapeutic targets. The targets were validated in the c9ALS Drosophila, or fruit fly, model, which mimics ALS. Of the 28, 18 were validated to have functional correlations to ALS, and eight previously unreported genes KCNB2, KCNS3, ADRA2B, NR3C1, P2RY14, PPP3CB, PTPRC, and RARA strongly rescued neurodegeneration through their suppression. 

“From AI-powered target discovery based on massive datasets, to biological validation by multiple model systems (fly, mouse, human iPS cells), to rapid clinical testing through investigator-initiated trials (IIT), these represent a new trend that may dramatically reduce the costs and duration and more importantly the success rate of developing medicines, especially for neurodegenerative diseases” said Bai Lu, PhD, Professor at Tsinghua University and Founder of 4B Technologies.

Feng Ren, Co-CEO and SCO of Insilico, said that the company is finding partners to take some targets into human trials for ALS. Beyond that, Insilico is also expanding PandaOmics to discover new targets for other disease areas such as oncology, immunology, and fibrosis, Ren added.

Profile of Insilico 

Founded in 2014, Insilico began building up its AI platform with a massive collection of data. Since its release in September 2020, PandaOmics has been licensed to nine of the top 20 pharmaceutical companies, according to 2020 revenue. 

In 2022, Insilico began a multi-asset partnership with Fosun Pharma and EQRX, as well as a drug discovery co-development deal with EQRX in March. Less than 40 days into the Fosun deal, Insilico managed to nominate a preclinical candidate for the QPCTL program for cancer immunotherapy, reaching its first milestone. 

Last month, the AI player raised $60 million in a Series D funding. The proceeds will be used for the expansion of its AI platform and advancement of its lead AI-designed Phase 1 program targeting fibrosis.

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Joy Lin

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