PTC Buys a Fabry Disease Gene Therapy From Bankrupt Sangamo for Up to $211M
The one-time gene therapy ST-920 is heading toward an FDA filing; PTC picked it up at auction after gene-medicine pioneer Sangamo filed for bankruptcy.
Yale researchers found that omega-3 and omega-6 fats can dampen natural killer cells’ tumor-fighting power in mice — via a receptor called LRP5. It’s early, and not a reason to change your diet.
The one-time gene therapy ST-920 is heading toward an FDA filing; PTC picked it up at auction after gene-medicine pioneer Sangamo filed for bankruptcy.
A large proteomics analysis of a landmark Parkinson’s study is now public, aiming to sort patients into subtypes and speed more targeted, biology-driven therapies.
Zipalertinib plus chemo so clearly slowed a hard-to-treat form of lung cancer that the trial was stopped early — setting up a fight with drugs from J&J and AstraZeneca.
Iberdomide is the first approved CELMoD — a next-generation protein degrader — cleared in a three-drug combination for multiple myeloma as early as first relapse.
Definium’s dissolving LSD tablet beat placebo in a large trial for generalized anxiety disorder, with effects seen within days — sending its shares up.
With more than 2,000 dead in what may become the second-largest Ebola epidemic ever, WHO is moving to test Merck’s Ervebo vaccine in a Phase 3 trial in Congo.
Retatrutide isn’t approved anywhere — but a gray market of compounders and ‘peptide’ sellers is already hawking copies, prompting Lilly to go to court.
Co-founded by Georg Schett — who showed CAR-T can drive lupus into remission — the Deerfield-backed startup aims to reset the immune system with more convenient antibodies.
Beyond hunger, drugs like Ozempic appear to dial down urges for alcohol, nicotine and more — and researchers point to a reward hub called the lateral septum.
A French randomized trial found healthy, low-risk people over 75 who stopped their preventive statins were no worse off on death or heart events over three years — with big caveats.
In lab-infected human immune cells, dexamethasone dialed down damaging inflammation from a stubborn lung infection while still letting the cells fight the bacteria.