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Sarepta's Gene Therapy Killed Children. The FDA Stopped Short of Pulling It.
The FDA slapped its most severe safety warning on Elevidys, Sarepta's gene therapy for Duchenne muscular dystrophy, after at least two children died from acute liver failure following treatment. Non-ambulatory patients are now locked out entirely. Sarepta simultaneously cut 500 jobs (36% of its workforce) to save $400 million annually, and its stock has seen extreme volatility, including a 35% single-day surge. The confirmatory trial had already failed, and the company is now betting on a modified immune suppression strategy that hasn't been proven yet.
Why it matters: This isn't just a Sarepta crisis. Elevidys is the only gene therapy on the market for DMD, and its safety failures are forcing the entire AAV gene therapy field to reckon with the dangers of high-dose systemic delivery, potentially reshaping trial designs, dosing strategies, and regulatory standards for years to come.
Read more →Deals and M&A
Lilly Bets $3.35 Billion on a Chinese Biotech Most People Have Never Heard Of
Eli Lilly agreed to pay up to $3.35 billion for access to InnoCare Pharma's drug discovery platform, targeting up to five undisclosed targets. InnoCare pockets about $100 million upfront; the rest is milestones. It's one of the largest China-to-Western licensing deals ever, and it lands amid a boom that saw 81 such deals worth $110 billion in the first half of 2026 alone.
Read more →Lilly Drops $2.9 Billion on a Startup With Zero Revenue
Merida Biosciences, with one Phase 1 drug and no sales, just got acquired by Lilly for up to $2.875 billion. The lead candidate targets Graves' disease and thyroid eye disease. It's the latest in Lilly's multi-billion-dollar immunology shopping spree as the company races to build growth engines beyond GLP-1.
Read more →Novo Nordisk Spends $1.4 Billion to Make Sure Its Future Isn't Stuck in a Needle
Following a billion-dollar deal with Nanexa for long-acting injectables, Novo signed a $1.4 billion partnership with Orbis Medicines to develop oral macrocycle drugs for cardiometabolic disease. Combined, that's over $2.5 billion committed to solving the delivery problem. The subtext: Eli Lilly's oral obesity pipeline is breathing down Novo's neck.
Read more →Clinical and Regulatory
What If Your Body Could Build Its Own Cancer Therapy? First Human Data Says Maybe.
IASO Bio's in vivo CAR-T therapy delivered a 90% response rate in ten multiple myeloma patients, with no need for cell collection, factory manufacturing, or lymphodepleting chemo. A single IV infusion reprogrammed T cells inside the body. It's only ten patients, but if the concept holds, it could dismantle the $400,000-per-treatment economics of cell therapy entirely.
Read more →The 40-Year 'Undruggable' Target Just Got Its First FDA-Approved Drug
Revolution Medicines is now shipping RASONQUE, the first multi-selective RAS inhibitor for metastatic pancreatic cancer, after FDA approval. The drug roughly doubled survival compared to chemo in a disease where 92% of tumors carry KRAS mutations that scientists couldn't touch for four decades. First-line trials are already underway, and the stock is trading around $205 with a consensus analyst target around $230.
Read more →Lilly Gets FDA Nod for a Precision Breast Cancer Combo That Hunts a Specific Mutation
The FDA approved Lilly's Inluriyo plus Verzenio for ER-positive, HER2-negative breast cancer carrying ESR1 mutations, which develop in 30-40% of patients whose tumors outsmart hormone therapy. The combo cut progression risk by 47%. It's a niche approval, but it gives Lilly a credible oncology foothold as it diversifies beyond obesity drugs.
Read more →Roche Takes a Swing at Progressive MS With an Enzyme Nobody Has Successfully Drugged
Roche revealed the science behind RG-6182, a first-of-its-kind reversible MAGL inhibitor now in Phase II for progressive MS. The enzyme breaks down the brain's natural protective cannabinoids while generating inflammatory byproducts. Blocking it addresses both problems at once, and in a disease with essentially one approved therapy for primary progressive MS, the playing field is wide open.
Read more →Policy and Industry
The White House Just Used a Budget Trick to Claw Back $1 Billion, and Biotech Is Watching
The Trump administration invoked a rare pocket rescission to cancel nearly $1 billion in approved spending, mostly from HHS. It didn't directly hit NIH this time, but it sets a precedent alongside proposed 41% NIH cuts and thousands of frozen grants. For an industry whose drug pipeline starts with federally funded university labs, the signal is ominous.
Read more →Lundbeck Ditches Suburban Chicago for Boston's Brain Belt
The Danish neuroscience company is relocating its entire U.S. headquarters from Deerfield, Illinois to Boston's Seaport district, joining other major pharma companies in the biotech capital. With Phase III programs in epilepsy, neurodegeneration, and migraine, Lundbeck wants closer access to the talent and partnership network it needs for its next decade.
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