March 18, 2025

The need-to-know this morning

Stoke Therapeutics CEO Ed Kaye is stepping down, the company said. He will be replaced on an interim basis by Director Ian Smith, a former Vertex Pharmaceuticals executive. A search for a permanent CEO is underway. Stoke is developing a treatment for a rare and severe form of epilepsy.
Sarepta Therapeutics said a patient with Duchenne muscular dystrophy died following treatment with its Elevidys gene therapy. The cause was acute liver failure.

biotech

CRISPR company Arbor gets more cash from ARCH

CRISPR company Arbor Biotechnologies said today that it raised a $73.9 million Series C led by ARCH Venture Partners, after it got regulatory clearance to start its first clinical trial in a rare disease affecting the liver and kidney.

That amount is a far cry from the $215 Million Series B that the company raised four years ago, when low interest rates fueled a gene-editing bubble. CEO Devyn Smith declined to comment on whether the biotech, which is spun out of Feng Zhang's lab, had to take a cut in valuation in this new round, but he implied it may have: "People can probably determine what most rounds are looking like these days."

Still, the new investment should give Arbor funding into 2027, providing it more time to collect data and begin proving out its thesis.

regulation

FDA's chaotic (and cramped) first day in the office

Picture this: You arrive to the office and there's a long line just to get into the building. You're scared to drink the water in the building since it tested positive for Legionella bacteria a few months ago. Some of your colleagues are assigned to work in conference rooms shared with dozens of other people. Another is assigned to work in a storage closet inside of a district courthouse, underneath jail cells.

This is what it looked on the first day back to the office for most FDA employees yesterday, after the Trump administration earlier this year ordered all government employees to return to work in person.

Since the pandemic, the FDA had been allowing employees to work from home to attract highly-skilled workers despite paying lower salaries than industry. According to a 2022 staff survey, about 64% of the agency's staff had some sort of remote work agreement.

Now, experts worry that this return-to-office mandate — coupled with the chaotic firings and rehirings led by Elon Musk — will lead to a staff exodus at FDA.

politics

Genetics expert is first NIH director to leave under Trump

Eric Green, the longtime director of the National Human Genome Research Institute, has abruptly left his role, according to two employees with knowledge of the situation and internal communications reviewed by STAT.

He's the first director of one of the NIH's 27 institutes to leave his position in President Trump's second term. His departure comes as the NIH is bracing for more layoffs, as well as a restructuring aimed at centralizing some of the agency's core functions. It remains unclear if Green's departure is a harbinger of more shakeups to the agency.

politics

The key figures to know in HHS

The Department of Health and Human Services is quickly filling out with allies of President Trump and Robert F. Kennedy Jr. My colleague Isa Cueto tells us about eight key people to know in the agency.

They include Hannah Anderson, HHS deputy chief of staff on policy. She was a health policy adviser to the Senate HELP Committee Republicans, primarily focused on prescription drug pricing and private health insurance.

There's also HHS principal deputy assistant secretary Shana Weir, who is perhaps best known for working to overturn the 2020 election results in Trump's favor.

pharma

Opinion: Don't ban direct-to-consumer drug ads

Politicians, including newly confirmed health secretary Robert F. Kennedy Jr., have long called for a ban on pharma TV ads, but banning them won't work as intended, Hal Rosenbluth, CEO of health tech company New Ocean Health Solutions, argues in a new opinion piece.

Just as as alcohol prohibition drove demand underground, banning DTC drug ads might push patients toward less reliable sources of medical information, like social media influencers, online forums, or even black-market drug channels, he said.

Instead, policymakers should focus on implementing stricter regulations on pharma ads to help consumers make more informed choices, such as using clearer language and being more transparent about side effects, Rosenbluth writes.