The Readout Loud

Just how many employees is Sarepta Therapeutics laying off? And why did the Food and Drug Administration reject Ultragenyx’s rare disease drug over manufacturing qualms? Ultragenyx CEO Emil Kakkis joins us to discuss what was in the rejection letter his company received late last week, and whether he still has confidence in FDA Commissioner Marty Makary. We also discuss Capricor’s drug rejection, and Sarepta’s decision to lay off hundreds of staffers, but give raises to some executives.

On this week’s episode of the Readout LOUD: A closer look at the NIH’s grant-cutting legal playbook, a not-so-transparent transparency push by the FDA commissioner, and another big biotech acquisition. Our colleague Anil Oza joins us to unravel a previously unseen memo he obtained from HHS that lays out the legal framework being used to justify the termination of NIH grants. The disclosure comes as legal fights over the legitimacy of these funding cuts are being challenged in multiple courts.

We invite STAT’s senior writer for infectious diseases, Helen Branswell, and Washington correspondent Chelsea Cirruzzo on to the podcast to discuss the latest news at the CDC. The agency is in the spotlight once again, after the Senate grilled the potential head of the agency at a confirmation hearing and its federal vaccine advisory committee met for the first time after Robert F. Kennedy Jr. fired its existing 17 members and installed a new, smaller panel. We also discuss the latest news in the life sciences, including data presented at last weekend’s American Diabetes Association research meeting. We also squeeze in some conversation about summer blockbusters. (Check out Adam’s "Jaws" Lego set here [https://www.lego.com/en-us/product/jaws-21350?cmp=KAC-INI-GOOGUS-GO-US_GL-EN-RE-SP-BUY-CREATE-MB_ALWAYS_ON-SHOP-BP-PMAX-ALL-CIDNA00000-PMAX-MEDIUM_PRIORITY&ef_id=CjwKCAjw3_PCBhA2EiwAkH_j4u6C641HtJ41-EzmzqMRdmL72emEwON_tAoUpOZALYXF5RNCDCqPgRoCnl4QAvD_BwE%3AG%3As&gad_campaignid=19930807088&gad_source=1&gbraid=0AAAAADESMXKJpP41IAXmfcGiqWlLOzkNq&gclid=CjwKCAjw3_PCBhA2EiwAkH_j4u6C641HtJ41-EzmzqMRdmL72emEwON_tAoUpOZALYXF5RNCDCqPgRoCnl4QAvD_BwE&s_kwcid=AL%21790%213%21%21%21%21x%21%21%2119930801844%21]).

We discuss the tragic news [https://www.statnews.com/2025/06/15/duchenne-sarepta-gene-therapy-elevidys-patient-death/] that a second teenage boy with Duchenne muscular dystrophy died after taking Sarepta Therapeutics' gene therapy, raising renewed questions [https://www.statnews.com/2025/06/16/duchenne-muscular-dystrophy-community-reacts-second-death-sarepta-therapeutics-elevidys/] about the FDA's standards for approving drugs. We look at the continued turmoil within the FDA, including the forced dismissal [https://www.statnews.com/2025/06/18/top-gene-therapy-regulator-forced-out-at-fda/] of top gene therapy regulator Nicole Verdun, as well as the announcement of a controversial drug-review voucher program [https://www.statnews.com/2025/06/17/fda-new-priority-reviews-will-cut-months-off-drug-approvals/] that seems to be steering the agency into politics. We also talk about a private equity firm's decision [https://www.statnews.com/2025/06/16/harvard-lab-to-be-financed-by-39-million-from-private-equity-firm-from-turkey/] to fund a research lab at Harvard University, filling a void created by government spending cuts. And we look at the approval [https://www.statnews.com/2025/06/18/fda-approves-gilead-hiv-prevention-drug-lenacapavir-yeztugo-next-best-thing-to-vaccine/] of a groundbreaking HIV prevention medicine from Gilead Sciences, as well as a recap of the big BIO convention this week and a preview of the upcoming American Diabetes Association meeting this weekend.

First, we chat about how pharma companies still don’t have much clarity on Trump’s most-favored nation drug pricing policy, biotech’s next big takeout target, and more. Then we bring on Kathryn Edwards, a vaccine expert and a former member of the CDC advisory panel, to parse through the turmoil.