CAR-T, China, Gene Editing

As gene editing explodes, a new report from Goldman says Chinese groups are seizing the lead on CRISPR and CAR-T studies

Contrary to what you might believe, biopharma companies in the US are not the leaders in clinical trials using gene editing tech. While a slate of US/European pioneers have been lining up their first human trials, investigators in China are already well ahead in testing CRISPR-edited cells in humans, according to a deep dive on gene therapies from Goldman Sachs analyst Salveen Richter.

“As of the end of February 2018,” Richter and her team reported, “there were nine registered clinical studies testing CRISPR-edited cells to treat various cancers and HIV infection in China, vs. only one study in the U.S. All of the studies were initiated / sponsored by top-tier public hospitals across China, and >80 patients were reported as being treated by these investigational genome medicines.”

Last year alone, Richter adds, the National Natural Science Foundation of China provided funding for more than 90 CRISPR projects — more than 270 over the last 4 years. With no regulations on these gene editing projects, hospitals in China in particular have been quick to accelerate their CRISPR work, a situation that might change soon if China’s main drug agency steps in and applies the brakes, as Goldman expects will happen.

And Chinese investigators are already rivaling the US in the total number of CAR-T studies that are being done. As of February, Goldman Sachs counted 153 CAR-T trials in China, compared to 164 in the US, 73 in Europe and 56 in all of the rest of the world combined.

The one company furthest out in front is Legend Biotechnology, which J&J paid $350 million to partner with as it entered the global race to develop new therapies in the wake of historic approvals for Kymriah (Novartis) and Yescarta (Gilead/Kite). And if these companies and trials hit pay-dirt data, as they promise to, Goldman believes that China will reap a considerable benefit by selling these therapies at a much lower rate than the pioneers on the market, spurring a significant amount of medical tourism.

What is this kind of market worth for the rivals looking to compete in it?

That depends on the supply of patients.

If your new genome medicine either promises to cure a disease or delay any further therapy for a lengthy period of time, notes the Goldman report, then your market could play out quickly as you race through the patient pool that is available. That’s what Gilead experienced when it came up with a painless cure for hepatitis C.

Not only did the drugs from Gilead shrink the pool, Goldman reports in a cold analysis of the numbers, they also reduced the number of carriers present to keep spreading the disease, further shrinking the market. From the report:

The potential to deliver “one shot cures” is one of the most attractive aspects of gene therapy, genetically-engineered cell therapy and gene editing. However, such treatments offer a very different outlook with regard to recurring revenue versus chronic therapies, particularly in certain diseases where it is possible to cure a large proportion of the prevalent patient pool (or at least prevent an additional dose from being required for an extended period). While this proposition carries tremendous value for patients and society, it could represent a challenge for genome medicine developers looking for sustained cash flow. Gilead is a case in point, where the success of its hepatitis C franchise has gradually exhausted the available pool of treatable patients. 

The most lucrative diseases for these new curative therapies, Richter adds, would be big fields like hemophilia or areas where there’s a large supply of new patients each year — like cancer. Spinal muscular atrophy, which afflicts a standard set of infants each year, is also viable. 

That may not be the kind of math that biopharma execs like to discuss in public, but it’s certainly the kind of equations they review carefully while deciding how to spend R&D budgets.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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