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Good morning to everyone except the Empire State Building.
Chinese biotechs advance CRISPR Duchenne drugs
As U.S. programs to develop treat treatments for Duchenne muscular dystrophy stall, two of the first efforts in China to treat the condition with CRISPR gene editing are taking off.
One biotech, GenAssist, has already dosed one young boy. Meanwhile, the other company, Huidagene, expects to dose the first of several boys in the next few weeks and report data by the first quarter of next year.
Scientists and advocates have long hoped that CRISPR could unlock a more powerful treatment for Duchenne than the technology used in Elevidys, the much-debated gene therapy from Sarepta. The need for such a treatment only grew last November, when trial results confirmed that Elevidys, while potentially useful, is far from a cure.
Read more from STAT’s Jason Mast.
How the FDA will regulate AI in drug development
The biopharma industry is increasingly using AI to try to make drug development more efficient. For example, AI models can predict which kinds of patients will respond the best to a drug. AI-derived biomarkers are being proposed as endpoints in human studies. And some groups are supplementing placebo groups with simulations.
But how will the FDA regulate trials that use AI? The agency is set to publish draft guidance on this topic by the end of the year. In the meantime, my colleague Katie Palmer interviewed Tala Fakhouri, co-lead of the AI Council recently established by the FDA’s Center for Drug Evaluation and Research.
They discussed the surge in drug submissions that incorporate AI, balancing the risk and benefits of using AI when evaluating applications, and how the FDA can keep up with the rapidly evolving technology.
Read more from STAT’s Katie Palmer.
Harris plan may mean more aggressive drug negotiation
Vice President Harris announced a proposal yesterday to use savings that Medicare gets from drug price negotiations to fund new home care, vision, and hearing benefits for older Americans.
The pharma industry’s fear when lawmakers first allowed Medicare to negotiate drug prices through the IRA was that once the firewall prohibiting Medicare from interfering with drug prices was broken, Congress would find expanding the program to pay for other policies irresistible. If Harris has her way, those fears could become reality.
Read more from STAT’s Rachel Cohrs Zhang and John Wilkerson.
Cuban’s anti-PBM agenda is working with Harris
As part of the above plan that was sent out to reporters yesterday, the Harris campaign also included a line proposing to crack down on PBMs to increase transparency around costs. It appears that Mark Cuban was given a heads up on it.
The business mogul hosted a roundtable with reporters yesterday when the proposal was sent out, and he indicated he knew the announcement was coming. “I think they’re also supposed to be talking about transparency and PBMs today,” he said.
The tip-off is a signal of Cuban’s ties to the Harris campaign. After launching Mark Cuban Cost Plus Drugs, a company that aims to lower drug costs by bypassing traditional middlemen, Cuban is now using his platform as a staunch, high-profile Harris advocate and informal campaign adviser to push his health care ideas.
Read more from STAT’s Rachel Cohrs Zhang.
AI biotech Insitro partners with Lilly in unusual deal
Insitro, a machine learning drug development company, has struck three agreements with pharma giant Eli Lilly for new medicines for metabolic diseases. But the deal is a little non-traditional: After identifying drug candidates, Insitro will be the one responsible for commercializing the drugs, and Lilly will be eligible for milestones and royalties.
Under one agreement, Insitro and Lilly will work to develop an antibody candidate for an unspecified metabolic disease. After the candidate has been identified, Insitro will be responsible for the remaining development and commercialization. Under the other two agreements, Insitro has the option to license Lilly’s N-acetylgalactosamine (GalNAc) delivery technology, which will help deliver two different small interfering RNA therapeutics to two different targets in the liver.
Read more from STAT’s Brittany Trang.
Prime Medicine lays off workers as it trims pipeline
From my colleague Jason Mast: Last month, Prime Medicine shelved a large portion of its gene-editing pipeline, as it looked to concentrate limited resources on getting a couple key programs into clinical trials. CEO Keith Gottesdiener declined at the time to say whether it would layoff staff as part of the reshuffling.
Prime has now confirmed to STAT that there were a “small number of people impacted.” It declined to say how many people were let go but said they primarily worked on the shelved programs, which encompassed conditions such as eye, brain, and muscle diseases. “There were no significant changes to headcount,” a spokesperson said.
At least a half-dozen gene-editing companies have laid off staff in the past year, as the market has soured on sector. After a peak-pandemic frenzy, investors have shown limited patience with companies whose technology might not pay off for years and have raised questions about how profitable one-time cures for rare diseases are.
Jim Wilson’s new company strikes deal with Brazil
From my colleague Jonathan Wosen: Gemma Biotherapeutics, a new company led by gene therapy pioneer Jim Wilson, and Brazil’s health ministry have announced a $100 million agreement to make gene therapies for rare disease accessible in Brazil.
The funding will help Gemma Bio develop therapies for six neurological diseases, including lysosomal storage disorders, a class of conditions that cause toxic waste to build up in nerve cells. In return, Fiocruz, the science and technology agency of Brazil’s health ministry, will have access to Gemma’s gene therapy manufacturing capabilities and will be responsible for supplying and distributing approved therapies in the country. Gemma will collect royalties on any sales.
The agreement should streamline the timeline for these drugs to reach patients and reduce their cost, Wilson said. It’s the first concrete example of how Gemma plans to make gene therapies globally accessible, and, he told STAT, likely not the last: “This will be one of, hopefully, a number of really important alliances.”
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