Embryo editing advances reignite ethical debates

Scientists have used a newer, more precise form of CRISPR in early human embryos to better understand the genes at play during the earliest stages of human development. The work, published in Nature, also suggests base editing is gentler than earlier versions of CRISPR, avoiding the chromosome damage that has raised safety concerns. Still, the embryos frequently ended up as mosaics, STAT’s Andrew Joseph and Megan Molteni write, with a mix of edited and unedited cells.

“If you are thinking about using this technology for clinical use, you need uniform editing,” Dieter Egli, a Columbia researcher, told STAT. “You cannot afford to have mosaicism.”

The study could intensify the debate over human embryo editing: While researchers say the work is aimed at answering fundamental biological questions and could improve fertility research, there’s deep concern that such advances could eventually pave the way toward designer babies.

washington

Cassidy targets 340B with sweeping overhaul

Sen. Bill Cassidy unveiled legislation that would significantly reshape the 340B drug discount program. This has long been a priority for the pharmaceutical industry, STAT’s John Wilkerson writes.

“Clearly, there are real transparency and oversight concerns that prevent 340B from translating to better access and lower costs for patients,” Cassidy said, adding that he considers the bill a starting point for policymaking.

The proposal would require many hospitals to share more of their discounted drug savings directly with low-income patients. They’d also have to limit the use of contract pharmacies and give drugmakers the option of paying rebates instead of providing upfront discounts. Hospitals, however, say that 340B revenue helps fund charity care and other essential services.

podcast

The mysterious case of Eli Lilly's obesity drug

Who got exclusive access to Eli Lilly's highly anticipated obesity candidate? Why are drugmakers spending so much money on acquisitions? And are hair loss drugs a good investment?

We discuss all that and more on this week's episode of “The Readout LOUD,” STAT's biotech podcast. We bring on our colleague Lizzy Lawrence to discuss her scoop that Lilly and the Food and Drug Administration have allowed one person to receive the pharma company's obesity candidate, retatrutide, through the FDA’s “compassionate use” program.

We also discuss the resurgence of M&A, Lilly's interest in a hair loss drug, and then the FDA's reversal on rare disease drugs.

BIOTECH

BIO attendees confront China, AI, and Washington

This year’s BIO conference made clear that biotech executives are increasingly focused on forces outside the lab. Industry leaders said they feel more confident navigating the Trump administration than they did a year ago, but remain deeply concerned about drug pricing policies. And, perhaps above all, there’s looming concern over the prospect of China overtaking the U.S. in pharmaceutical innovation.

Artificial intelligence has also entered a more pragmatic phase: Rather than asking whether AI belongs in drug discovery, companies are now trying to identify where it delivers measurable value while managing soaring costs.

Read more for takeaways from the conference.

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