Warum lassen sich Pharma-Riesen in der Schlange auf eine Zusammenarbeit mit der chinesischen Frau-CEO, der härtesten in der Silicon Valley AI-Szene, einreihen?
Remember the "Ice Bucket Challenge" that took the world by storm in the summer of 2014?
As CEOs, movie and TV stars, and ordinary citizens poured buckets of ice water over their heads and felt the moment of "freezing," this phenomenal online event also brought a cruel disease - amyotrophic lateral sclerosis (ALS), commonly known as "Lou Gehrig's disease" - into the public eye on such a large scale for the first time.
This is a real medical "incurable disease." Patients gradually lose control of their muscles while their consciousness helplessly watches as their body "freezes" bit by bit until they finally suffocate.
For over a century, ALS has remained the "graveyard" in new drug development - the failure rate in development is over 90%, and it can easily take a decade and billions of dollars before a new drug hits the market. Even the world's leading pharmaceutical companies keep failing here and don't know what to do.
Yet, in this area declared almost "unsolvable," a young Chinese entrepreneur - Alice Zhang - and her company Verge Genomics are trying to tackle this "impossible" task with a completely new weapon - AI + human genomic data.
What exactly makes this Chinese-founded AI startup in Silicon Valley special? How is its work on this century-old problem going? Where are its advantages compared to local biotech giants?
Today, let's take a look at the story of Verge Genomics and find the answers to these questions.
01
With an "incurable disease" like ALS, the traditional approach has obviously hit a dead end. To get out of this situation, one must choose a completely new approach. Verge Genomics' answer is both bold and rational.
The core idea: "All-in-Human", finally saying goodbye to the mouse
Let's first pose a question that has been plaguing scientists in drug development for decades: Why do drugs work so well in mice but so often fail in human clinical trials?
This is the famous "graveyard" of development. Countless promising drug candidates fail here, costing pharmaceutical companies billions of dollars in investments and a decade of valuable time and repeatedly dashing patients' hopes.
The root of the problem lies in the excessive reliance on animal models.
Alice Zhang, the founder of Verge Genomics, and her team believe that the genetic differences between humans and mice are huge. If you use the wrong model to solve the right problem, you've taken the wrong path from the start.
So, they've developed a revolutionary core idea - "All-in-Human" (driven by human data).
Put simply, you completely skip the intermediate step of animal testing and let artificial intelligence directly learn about human diseases in the real world from the start.
To implement this idea, they've developed the company's core technology - the CONVERGE® platform.
This platform does only one thing: it extracts multi - dimensional biological data, including genomic and transcriptomic data, from a huge amount of real tissue samples from the brains and spinal cords of human patients (including deceased donors) and "feeds" this data to the powerful AI.
It's as if we're not giving the AI a wrongly translated "mouse disease manual" but directly a high - resolution original manual on human diseases to cure a human's illness.
By learning from a huge amount of data from real patients, the AI can find the gene targets that are truly related to the disease with unprecedented accuracy and predict which drugs can act on these targets. This fundamentally improves the success rate and efficiency of drug development.
And who came up with this revolutionary idea? Not a gray - haired, experienced pharma expert, but a young Chinese entrepreneur - Alice Zhang.
Her resume is a model of an outstanding student: she completed her bachelor's degree in molecular biology at Princeton University and then joined the prestigious MD/PhD program (Doctor of Medicine/Doctor of Philosophy) at UCLA - Caltech.
If she had followed this brilliant academic path, she could have become an excellent doctor or scientist. But at this time, she noticed a big "gap": on the one hand, the rapidly advancing AI and big - data technologies, and on the other hand, the slowly advancing and narrowly closed traditional biological laboratories. There seemed to be an invisible wall between them.
She wasn't satisfied with just publishing papers in the academic world. She wanted to tear down this "wall" and use the latest technologies to solve the most difficult medical problems.
So, she made a bold decision: she left the doctoral program at the top university and founded Verge Genomics in 2015.
One could say that Alice Zhang's "cross - over" genetics was integrated into Verge's DNA from the start. She understands both the latest biological sciences and AI and big data. It's precisely this unique perspective that allows her and her team to break the rules and climb the seemingly insurmountable mountain range of "Lou Gehrig's disease" with new logic.
02
In Silicon Valley, a revolutionary idea might help you get funding, but only hard results can earn you real respect among a table full of giants. This is especially true for Verge Genomics, which operates in the "life - threatening" new drug development industry.
So, how is this Chinese - founded AI company doing? The answer lies in its research and development pipeline and its "circle of friends."
Milestone pipeline: From 0 to the clinic in just 4 years
The most important criterion for evaluating a biotech company is the progress of its drug development pipeline. Verge has achieved an amazing performance here.
Verge's leading project is a drug candidate named VRG50635, which targets exactly the century - old problem we mentioned at the beginning - "Lou Gehrig's disease" (ALS).
The most impressive thing is its speed: From the discovery of a new drug target by AI in a huge amount of human gene data to the development of a drug candidate and its initiation into human clinical trials, Verge only took 4 years.
What does this mean? In the traditional pharmaceutical industry, it takes an average of over 12 years to go through this process. Verge has shortened this period by two - thirds. This is not only a victory of time but also the ultimate confirmation of the predictive ability and efficiency of its AI platform CONVERGE®. Currently, VRG50635 has successfully entered the clinical phase and is now being tested for its safety and effectiveness in real patients, giving new hope to millions of patients worldwide.
Even more importantly, Verge's platform is not just a one - time success. It has quickly transferred the ability to "find drugs with AI" from the "hard nut" of neuroscience to another globally watched blue - ocean market - obesity and metabolic diseases.
Its second drug candidate, VRG201, was specially developed for this purpose and is currently in the last phase before clinical trials (IND - enabling). This proves that Verge's technology platform has strong scalability. It's not a "specialist" that can only solve a single problem but a "universalist" that can support multiple disease areas.
If the progress speed of its own research and development pipeline shows Verge's "inner strength," its glittering "circle of friends" represents the vote of confidence from the best players in the industry, who are voting with "real gold."
These pharma giants have the world's best scientific teams and the strictest evaluation criteria. When they decide to support a young company, it means that the company's technology has passed the strictest tests.
Partnership with Eli Lilly: In 2021, Verge entered into a partnership with the world - leading pharmaceutical company Eli Lilly to develop a new therapy for "Lou Gehrig's disease." This partnership not only brought upfront funds for Verge but also subsequent milestone payments and potential sales royalties worth billions of dollars. This is equivalent to Eli Lilly acknowledging that Verge's AI platform can find treasures that even it itself can hardly discover.
Partnership with AstraZeneca: After Eli Lilly, another world - leading pharmaceutical company, AstraZeneca (through its subsidiary Alexion), has also extended an invitation to Verge. Both sides have agreed to use Verge's AI platform to find new targets for rare neurodegenerative diseases and neuromuscular diseases.
The significance of these top partnerships goes far beyond money. It's the strongest recommendation to the world: Verge Genomics not only has an exciting story to tell but also a powerful engine, confirmed by the industry giants, that can continuously deliver high - quality results.
03
In Silicon Valley, Verge Genomics doesn't fight alone. The race for AI - based drug development is already populated by many players, including Insitro, founded by Stanford's "AI goddess" Daphne Koller, and Recursion Pharmaceuticals, which has already successfully gone public. Like Verge, they also use AI to overcome the traditionally long and expensive model of drug development.
So, what's the special feature of Chinese AI companies, represented by Verge Genomics, when competing with each other? How does it build its own moat?
1. Strategy comparison
All AI - based drug companies have jointly recognized that the traditional development model is inefficient. But the ways to solve this problem are different.
The common way in the United States: Take Insitro as an example. It conducts extensive high - throughput experiments on induced pluripotent stem cells (iPSC) in a laboratory to generate a huge amount of data for training the AI model. This is undoubtedly a step forward as it breaks away from animal models, but the data essentially comes from an "in - vitro" experimental environment.
Verge's different advantage: Verge's "all - in - human" strategy is even more radical and decisive. It directly chooses the origin closest to the target - real tissue samples from human patients. By analyzing a huge amount of human brain and spinal cord tissue data...