Original reporting and analysis on the stories shaping biotech.
Four years of data show Stoke and Biogen's zorevunersen doesn't just reduce seizures in Dravet syndrome kids; it may actually improve their cognition. If the Phase 3 confirms it, this could rewrite the playbook for an entire class of genetic diseases.
Pfizer just sold a clinical-stage cancer drug from its $43 billion Seagen acquisition to a tiny Canadian biotech for $12 million. The deal says a lot about the growing gap between what Pfizer paid for Seagen and what it's getting back.
The FDA is letting four AI medical devices treat real patients before proving they actually work. It's called the TEMPO pilot, and it might be the biggest regulatory experiment in modern healthcare.
A Chinese biotech's drug just beat the world's best-selling medicine, Keytruda, in a head-to-head survival study for lung cancer. Merck's $32 billion franchise has a real challenger for the first time, and the implications are enormous.
For 77 years, Alexander disease had zero approved treatments. Ionis Pharmaceuticals just changed that with Zanvastro, and the implications stretch far beyond one ultra-rare condition.
AbbVie just dropped Phase 3 data for its myeloma bispecific, etentamig, and the numbers are hard to ignore: 74% response rate and a 60% cut in disease progression. The catch? Three competitors already have approved drugs on the shelf.
Every blockbuster weight-loss drug has a dirty secret: they burn muscle along with fat. Roche just paid $190 million for a completely new type of obesity drug that might fix that, and the preclinical data is turning heads.
An Indian pharma company just licensed a Chinese-developed Keytruda copycat for the U.S. market, creating an unlikely supply chain aimed at the world's bestselling drug. With $32 billion in annual sales and patents starting to crack, the race to disrupt oncology's biggest cash cow is heating up.
A mouse study published in Science found that psilocybin given before chemotherapy prevented nerve damage for up to 8 months. With zero approved drugs to prevent chemo-induced neuropathy and over half of patients affected, this preclinical finding could reshape how oncologists think about one of cancer treatment's nastiest side effects.
The team that built MyoKardia (acquired by Bristol-Myers Squibb for $13.1 billion) just took their new cardiac biotech public in a $400 million upsized IPO. Three late-stage drugs, $554 million in prior venture funding, and a stock that popped on day one: Kardigan is either biotech's next blockbuster or its most expensive sequel.
Three patients with autoimmune diseases died in Novartis CAR-T trials, forcing the company to pause eight studies and the entire field to confront an uncomfortable question: can cancer's most powerful therapy ever be safe enough for diseases that aren't trying to kill you?
The FDA told uniQure its Huntington's disease gene therapy data weren't good enough to file for approval. Then the agency reversed course. Now AMT-130 could become the first treatment that actually slows a disease with zero disease-modifying options.
GSK is paying up to $1.3 billion for a HUTCHMED cancer drug that hasn't been tested in a single human. The molecule targets both KRAS and EGFR simultaneously, attacking the biological escape route that makes these cancers so hard to treat.
Novartis just signed a deal worth up to $5.2 billion with a Chinese biotech most people have never heard of. The target: next-generation heart drugs built on gene-silencing technology, and it's part of an aggressive cardiovascular shopping spree that's reshaping the company's future.
A bispecific antibody just beat Keytruda in an overall survival showdown for first-line lung cancer. With nearly $30 billion in annual Keytruda sales at stake, this could be the biggest competitive threat Merck has ever faced in oncology.
ARPA-H just gave $54.5 million to a six-company team trying to squeeze an entire mRNA factory onto a lab bench. If their continuous-flow system works, it could unlock personalized medicines that today's batch manufacturing can't touch.
The FDA is cracking down on clinical trial data from China, expanding inspections and demanding earlier verification of foreign study sites. With roughly half of the world's innovative-drug trials now touching China, the policy shift could force dozens of companies to overhaul their development strategies.
Two deaths in China from CRISPR therapies delivered by viral vectors are forcing the gene-editing field to confront an uncomfortable truth: the editing tool works, but the delivery system can kill. The fallout is already reshaping regulations from Beijing to Washington.
Teva's experimental antibody just aced a mid-stage celiac disease trial, posting the best data anyone has seen in the IL-15 space. With zero approved targeted therapies and a market approaching $1 billion, the race to deliver the first real celiac drug is officially on.
A personalized cancer vaccine just posted 100% disease-free survival at three years in one of oncology's toughest cancers. Sixteen patients, zero relapses, and a field watching very closely.
Ultragenyx's Angelman syndrome drug showed zero benefit over sham treatment in a 129-patient Phase 3 trial, sending shares down over 40%. For families who'd pinned their hopes on the most advanced therapy in development, the fallout is devastating.
Eli Lilly has blown through $25 billion on biotech acquisitions this year, returning cash to 50+ venture investors and spinning the capital flywheel that funds the next wave of startups. But the recycled money isn't going where you'd expect.
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