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How Verge Genomics Uses AI To Tackle Parkinson’s, Alzheimer’s And ALS

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Verge Genomics is taking a novel approach to speed drug discovery for devastating neurodegenerative diseases such as Parkinson’s, Alzheimer's and ALS. Rather than spending exhaustive time with animal testing, the San Francisco-based company goes straight to the source. 

“To succeed in humans, you need to start with humans,” says CEO Alice Zhang, whose drug discovery combines artificial intelligence with human genomics. Animal testing can be successful in predicting success for some drugs, but when it comes to neurodegenerative diseases, human brains are far more complex. To keep human patients at the center of the drug discovery process, Zhang’s company has built one of the largest databases of brain tissue sequences in the world, with tissue from more than 1,000 human brains. 

Launched in 2015 and with $54 million in funding, Verge Genomics is one of several drug companies on the Forbes AI 50 list for 2021. The company sets itself apart from others in the AI drug discovery market, expected to grow to $2.1 billion by 2027, by decoding the DNA of patients who die of neurodegenerative diseases and using a machine learning algorithm to find genes that could serve as targets for new drugs.

Most biotech companies, Zhang says, look for potential treatments that target a single gene involved in a disease to be turned on or off and thus alleviate symptoms. By contrast, Verge Genomics looks for “master switches”—one gene that controls a set of important genes. “These help us find signatures that help treat say 90% of the patient population as against say just 5%,” she adds. Once they have found a target, the company’s in-house team designs drugs that can specifically interact with it. 

Zhang, who’s also a Forbes 30 Under 30 alum, says her company’s new drug candidate for ALS has shown great promise in the lab when tested in brain cells derived from patients. “The drug has shown to slow the death of these patients’ neurons and at times restore them back to the same levels as that of healthy people.” The drug will enter human clinical trials next year, she adds. 

A Virginia native, Zhang says that her parents, who immigrated from China, emphasized education when she was growing up. As a teenager,  she took a trip to China. It wasn’t a vacation, however. She was working with AIDS patients with a non governmental organization, and the experience left a lasting impact. “That was the first time I saw people facing death and the impact that lack of healthcare can have on human life,” Zhang says.

The idea to start looking at genes with AI to find cures for neurodegenerative diseases took shape when Zhang was a graduate student at UCLA. In one of her experiments, she had predicted a drug target for a nerve injury using genomics data. She injected injured mice with the drug candidate and it worked. “It was really one of the first drugs that came out from the platform,” Zhang says. 

AI has become a hot commodity in biotech, and several other companies listed on the Forbes AI 50 are also using it in innovative ways. For example, Burlingame, California based Genesis Therapeutics is looking for new drugs by using AI algorithms to explore the chemistry of molecules that could be used for potential therapies.  Another startup on the list, San Francisco, California based Atomwise, scans through billions of potential drugs and models their interactions with various proteins in an attempt to predict which might make for safe and effective treatments.   

Investors say Verge’s proprietary genomic data from human brains is unlike any other company in the field. “It sounds like zombies, getting access to brains,” says Emily Melton, founder of Threshold Ventures, which led Verge’s series A. “Finding proprietary data like Verge’s is really hard in neuroscience. Neurodegenerative diseases are a huge problem, which is only getting worse. In future, it is going to be one of the most critical trends which we will need to address.” 

Zhang says her journey from a nerdy teenager to a “non-traditional” CEO and cofounder hasn’t been an easy one; rather, it was full of rejections. “The startup ecosystem in biotech at least now is geared more towards these traditional experienced CEOs where a lot of the investment goes.” A lot of those people, she adds are experienced older male CEOs. 

Her dogged nature and unique approach, however, impressed Melton, which is why she invested in Verge. “Alice just really impressed me. She has raw intellect, insane drive and a growth mindset.” 

With her company’s first drug about to go through the clinical pipeline, Zhang is already  dreaming of expanding Verge Genomics’ presence beyond the realm of neuroscience.e “One day,” she says. “We will extend to diseases which are challenged by disease complexities and there aren’t available treatments for patients.”

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