From data to drugs: How AI is revolutionising drug development

The laboratory at Terray Therapeutics is a symphony of miniaturised automation. Robots whir, shuttling tiny tubes of fluids to their stations. Scientists in blue coats, sterile gloves and protective glasses monitor the machines.

But the real action is happening at the nanoscale. Proteins in solution combine with chemical molecules held in minuscule wells in custom silicon chips that are like microscopic muffin tins.

Every interaction is recorded, millions and millions each day, generating 50 terabytes of raw data daily – the equivalent of more than 12,000 movies.

The lab, about two-thirds the size of a football field, is a data factory for artificial-intelligence- assisted drug discovery and development in Monrovia, California, United States. It’s part of a wave of young companies and startups trying to harness AI to produce more effective drugs, faster.

The companies are leveraging the new technology – which learns from huge amounts of data to generate answers – to try to remake drug discovery.

They are moving the field from a painstaking artisanal craft to more automated precision, a shift fuelled by AI that learns and gets smarter.

“Once you have the right kind of data, the AI can work and get really, really good,” said Jacob Berlin, co-founder and CEO of Terray.

Most of the early business uses of generative AI, which can produce everything from poetry to computer programs, have been to help take the drudgery out of routine office tasks, customer service and code writing. Yet drug discovery and development is a huge industry that experts say is ripe for an AI makeover.

AI is a “once-in-a-century opportunity” for the pharmaceutical business, according to consulting firm McKinsey & Co.

Just as popular chatbots like ChatGPT are trained on text across the Internet and image generators like Dall-E learn from vast troves of pictures and videos, AI for drug discovery relies on data. And it is very specialised data – molecular information, protein structures and measurements of biochemical interactions. The AI learns from patterns in the data to suggest possible useful drug candidates, as if matching chemical keys to the right protein locks.

Because AI for drug development is powered by precise scientific data, toxic “hallucinations” are far less likely than with more broadly trained chatbots. And any potential drug must undergo extensive testing in labs and in clinical trials before it is approved for use by patients.

Companies like Terray are building big high-tech labs to generate the information to help train the AI, which enables rapid experimentation and the ability to identify patterns and make predictions about what might work.

Generative AI can then digitally design a drug molecule. That design is translated, in a high-speed automated lab, to a physical molecule and tested for its interaction with a target protein. The results – positive or negative – are recorded and fed back into the AI software to improve its next design, accelerating the overall process.

While some AI-developed drugs are in clinical trials in the United States, it’s still early days.

“Generative AI is transforming the field, but the drug-development process is messy and very human,” said David Baker, a biochemist and director of the Institute for Protein Design at the University of Washington.

Drug development has traditionally been an expensive, time- consuming, hit-or-miss endeavour. Studies of the cost of designing a drug and navigating clinical trials to final approval vary widely. But the total expense is estimated at US$1bil (RM4.71bil) on average.

It takes 10-15 years. And nearly 90% of the candidate drugs that enter human clinical trials fail, usually for lack of efficacy or unforeseen side effects.

The young AI drug developers are striving to use their technology to improve those odds, while cutting time and money.

Their most consistent source of funding comes from the pharma giants, which have long served as partners and bankers for smaller research ventures.

Today’s AI drugmakers are typically focused on accelerating the preclinical stages of development, which have conventionally taken four to seven years. Some may try to go into clinical trials themselves. But that stage is where major pharma corporations usually take over, operating the expensive human trials, which can take another seven years.

For established drug companies, the partner strategy is a relatively low-cost path to tapping innovation.

“For them, it’s like taking an Uber to get you somewhere instead of having to buy a car,” said Gerardo Ubaghs Carrion, a former biotech investment banker at Bank of America Securities.

The major pharma companies pay their research partners for reaching milestones towards drug candidates, which can reach hundreds of millions of dollars over years. And if a drug is eventually approved and becomes a commercial success, there is a stream of royalty income.

Companies like Terray, Recursion Pharmaceuticals, Schrodinger and Isomorphic Labs are pursuing breakthroughs. But there are, broadly, two different paths – those that are building big labs and those that aren’t.Isomorphic, the drug discovery spinout from Google DeepMind, the tech giant’s central AI group, takes the view that the better the AI, the less data that’s needed. And it is betting on its software prowess.

In 2021, Google DeepMind released software that accurately predicted the shapes that strings of amino acids would fold into as proteins. Those 3D shapes determine how a protein functions. That was a boost to biological understanding and helpful in drug discovery, since proteins drive the behaviour of all living things.

Last month, Google DeepMind and Isomorphic announced that their latest AI model, AlphaFold 3, can predict how molecules and proteins will interact – a further step in drug design.

“We’re focusing on the computational approach,” said Max Jaderberg, chief AI officer at Isomorphic. “We think there is a huge amount of potential to be unlocked.”

Terray, like most of the drug development startups, is a byproduct of years of scientific research combined with more recent developments in AI.

Berlin, who earned his doctorate in chemistry from Caltech, has pursued advances in nanotechnology and chemistry throughout his career. Terray grew out of an academic project begun more than a decade ago at the City of Hope cancer centre near Los Angeles, where Berlin had a research group.

Terray is concentrating on developing small-molecule drugs, essentially any drug a person can ingest in a pill, like aspirin and statins. Pills are convenient to take and inexpensive to produce.

Terray’s sleek labs are a far cry from the old days in academia when data was stored on Excel spreadsheets and automation was a distant aim.

“I was the robot,” recalled Kathleen Elison, a co-founder and senior scientist at Terray.

But by 2018, when Terray was founded, the technologies needed to build its industrial-style data lab were progressing apace. Terray has relied on advances by outside manufacturers to make the micro-scale chips that Terray designs. Its labs are filled with automated gear, but nearly all of it is customised – enabled by gains in 3D printing technology.

From the outset, the Terray team recognised that AI was going to be crucial to make sense of its stores of data, but the potential for generative AI in drug development became apparent only later – though before ChatGPT became a breakout hit in 2022.

Narbe Mardirossian, a senior scientist at Amgen, became Terray’s chief technology officer in 2020 – in part because of its wealth of lab-generated data.

Under Mardirossian, Terray has built up its data science and AI teams and created an AI model for translating chemical data to maths, and back again. The company has released an open-source version.

Terray has partnership deals with Bristol Myers Squibb and Calico Life Sciences, a subsidiary of Alphabet, Google’s parent company, that focuses on age- related diseases. The terms of those deals are not disclosed.

To expand, Terray will need funds beyond its US$80mil (RM376.6mil) in venture funding, said Eli Berlin, Jacob Berlin’s younger brother.

He left a job in private equity to become a co-founder and the startup’s chief financial and operating officer, persuaded that the technology could open the door to a lucrative business, he said.

Terray is developing new drugs for inflammatory diseases, including lupus, psoriasis and rheumatoid arthritis. The company, Jacob Berlin said, expects to have drugs in clinical trials by early 2026.

The drug-making innovations of Terray and its peers can speed things up, but only so much.

“The ultimate test for us, and the field in general, is if, in 10 years, you look back and can say the clinical success rate went way up and we have better drugs for human health,” Berlin said. – The New York Times