Brainstorm Health: Gene Editing Arms Race, Pfizer Duchenne Drug, Concentrated Caffeine

April 13, 2018, 6:44 PM UTC

Happy Friday, readers! This is Sy.

In recent years, the advent of gene editing and cell-engineering technologies to fight rare diseases, cancer, and a number other serious conditions has taken the biopharma world by storm across the globe. The Food and Drug Administration (FDA) approved therapies known as “CAR-T”, from Novartis and Gilead/Kite Pharma, that re-engineer patients’ own immune cells to destroy blood cancers last year. Chinese scientists launched the first known human trials of CRISPR—the genomic tech that involves slicing and dicing the body’s very source code—to combat cancer back in late 2016.

That latter development led renowned scientist and cancer immunotherapy expert Dr. Carl June to predict “Sputnik 2.0, a biomedical duel on progress between China and the United States” on this genomic front. But China is currently winning that race, outpacing the U.S. when it comes to developing new wave CAR-T and CRISPR treatments, according to a new Goldman Sachs analysis reported by Endpoints News.

“As of the end of February 2018, 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.,” wrote Goldman analyst Salveen Richter and her team in a research note. “All of the studies were initiated / sponsored by top-tier public hospitals across China, and [more than] 80 patients were reported as being treated by these investigational genome medicines.”

China is still a bit behind the U.S. on CAR-T trials, according to Goldman, but is hot on America’s trail. And when it comes to CRISPR, the nation has a distinct head start: China’s Natural Science Foundation funded 90 CRISPR initiatives in 2017 alone and nearly 300 in the past four years.

None of this means the U.S. is doomed to fall behind its international rival over the long run. For instance, stricter regulatory scrutiny in China could slow the roll on certain gene-editing and CAR-T projects. And there’s plenty of interest from pioneering biotechs and pharmaceutical partners in America to keep the field competitive. Down the line, though, issues like pricing could play an important factor in who wins the battle for market share around the globe since such treatments are extraordinarily expensive.

The issue is likely here to stay. Earlier this week, pharma giant Novartis struck a massive deal to purchase gene therapy developer AveXis for $8.7 billion, and manufacturing sites across the world are preparing for a big demand spike for groups that can carry out this kind of genetic and biological tinkering on a mass scale.

Read on for the day’s news, and have a fantastic weekend!


The data breach plague comes to an oxygen device maker. Digital delinquency is an all-purpose scourge. Hackers hit insurers, hospitals, credit monitoring agencies, financial titans, and pretty much any organization they can successfully target on a regular basis. And that includes more under-the-radar parts of the medical industry—such as Inogen Inc, an oxygen device maker that reported on Friday a data breach that affected 30,000 current and former customers who rented the company's products. Affected data included names, contact details, Medicare identification numbers, and insurance policy information, but no payment details, according to the company. (Reuters)


Pfizer is developing a muscular dystrophy gene therapy. Speaking of gene therapy... Viagra maker Pfizer has launched a (super) early stage gene therapy trial to treat Duchenne muscular dystrophy, a muscle-wasting disease that affects mostly boys and leaves most dead by their 20s. The trial stems from Pfizer's 2016 acquisition of Bamboo Therapeutics. Duchenne has been a growing interest area for rare drug makers since Sarepta's landmark approval its own drug; giants like Pfizer are now joining the fold. (Boston Business Journal)


The FDA goes after... Too much caffeine. The Food and Drug Administration (FDA) is turning its sights on bulk, concentrated caffeine products that could potentially be deadly. Such products should are illegal and should be yanked off the market, according to the FDA. "These products present a significant public health threat because of the high risk that they will be erroneously used at excessive, potentially dangerous doses," said the FDA said in a statement. "Highly concentrated and pure caffeine, often sold in bulk packages, have been linked to at least two deaths in otherwise healthy individuals." (NBC News)


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