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Intellia's CRISPR Trials Hit a Wall After Severe Liver Toxicity
The biggest in vivo CRISPR program in history just got benched. Intellia paused two late-stage trials for its gene-editing therapy nex-z after a patient in his 80s developed grade 4 liver toxicity, the most severe classification short of death. The FDA slapped clinical holds on both studies, which were enrolling roughly 1,200 patients across dozens of global sites. Holds have since been lifted with stricter safety protocols, but the episode is the second serious liver event in the program. Shares dropped 17%, and analysts slashed price targets across the board.
Why it matters: This isn't just an Intellia problem. Two liver toxicity cases in the same CRISPR program force the entire in vivo gene-editing field to confront an uncomfortable question: can you safely rewrite human DNA inside a living body at scale, or does the margin for error remain too thin for prime time?
Read more →Clinical and Regulatory
A Kidney Disease With No Good Options Just Got Another FDA-Approved Drug
Three years ago, IgA nephropathy patients had basically one targeted treatment. Now the options keep growing. The FDA granted Vera Therapeutics' Trutakna, a first-of-its-kind dual BAFF/APRIL blocker, accelerated approval. The drug cut protein leakage (a key marker of kidney damage) by 46% versus 7% for placebo. Analysts project $2.5 billion in peak sales by 2033. The catch: it's an accelerated approval, so long-term kidney protection data still needs to come through.
Read more →AstraZeneca's Amyloidosis Drug Flops, but the Company Isn't Giving Up
AstraZeneca's anselamimab missed its primary endpoint in a Phase 3 AL amyloidosis trial. The drug didn't reduce deaths or hospitalizations in the overall population. But AZ is pointing to a prespecified subgroup of kappa light-chain patients where survival improved 62%. Regulators typically treat subgroup wins in failed trials as hypothesis-generating, not proof, so a new focused trial is likely needed.
Read more →Kailera's Oral Obesity Pill Hits Its Goals in Two China Phase 3 Trials
Kailera's once-daily obesity pill delivered 11% weight loss over 44 weeks and beat a standard diabetes drug on blood sugar reduction. Solid numbers, but GI side effects were notable. Global Phase 2 data at higher doses is expected in 2027, with the oral GLP-1 market projected to hit $22 billion by 2030.
Read more →Deals and Strategy
From Lawsuit to Love Story: Novo Nordisk and Hims Team Up on Wegovy
Less than a year after suing Hims & Hers for patent infringement over knockoff semaglutide, Novo Nordisk is now selling branded Wegovy and Ozempic on the Hims platform. Hims agreed to stop mass-marketing compounded GLP-1 drugs; Novo gets access to Hims's subscriber base. Hims shares surged over 50% on the news. The deal signals that the era of cheap compounded semaglutide is winding down fast.
Read more →Lilly Pays $2.3 Billion for Ajax's Phase 1 Blood Cancer Drug
Eli Lilly is spending up to $2.3 billion on Ajax Therapeutics, whose only asset is a Phase 1 JAK2 inhibitor with 23 patients enrolled. The drug uses a novel "Type II" mechanism that locks JAK2 in its inactive form, and early data showed 21 of 23 patients had shrinking cancer-driving mutations. Lilly was a founding investor in Ajax, making this less a blind bet and more a calculated escalation.
Read more →Industry Watch
The FDA Is Hemorrhaging the Staff Who Approve Your Drugs
CDER, the office that greenlights every new drug in America, lost over 1,000 employees in fiscal 2025 and has cycled through five directors in roughly a year. More than half of senior leaders from a year ago are gone. With application volume climbing and a hiring freeze limiting replacements, cell and gene therapy rejection rates have already jumped from 18% to 38%.
Read more →Ionis Turns a Rare Disease Drug Into a $3 Billion Opportunity
Ionis just expanded Tryngolza's FDA label from a tiny genetic condition affecting thousands to severe hypertriglyceridemia, which hits nearly 3 million U.S. adults. In trials, the drug cut pancreatitis episodes by 85% and reduced triglycerides by up to roughly 72%. Peak sales projections jumped from over $1 billion to over $3 billion, marking Ionis's transformation from biotech's favorite licensor into a genuine commercial-stage company.
Read more →America's Only Syphilis Drug for Pregnant Women Just Disappeared
Pfizer's Bicillin L-A, the sole FDA-approved injectable penicillin for syphilis in pregnancy, has been in shortage since a recall over defective syringe stoppers. Full supply recovery isn't expected until late 2026 or 2027. With congenital syphilis cases up 700% since 2015 and no proven alternative for pregnant women, states are rationing a drug that's been saving lives since the 1940s.
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