Innate keeps developing the drug; Sobi takes commercialisation on accelerated approval. A structure that lets a research-stage company reach patients without building a sales force.
A modified vitamin A that behaves normally except in the one reaction that causes the damage. Seven years of patient follow-up, and a readout still three years away.
Two compounds that do opposite things to the same protein family. Neither will ever be given to a patient — that is not what they are for.
A $121 billion company’s quarter beat estimates. The stock story is a $2.3 billion rival with a sweat chloride readout coming.
Abdominal obesity alone: 47% higher mortality risk. Vitamin D deficiency alone: up to 91%. Both together: 123% — more than either, and the researchers propose a reason the two interact.
A sodium channel that exists almost only in pain-sensing nerves is why a non-addictive painkiller is chemically possible at all.
Same enzyme as cyclosporine, different binding site, and deliberately kept out of the brain. The point is not a new target — it is removing the toxicity.
A onetime genetic-medicine pioneer went bankrupt, and its Fabry gene therapy went to auction. PTC paid $111 million upfront for an asset heading into an FDA submission.
GLP-1 drugs strip muscle along with fat, and the industry has decided that is the next problem to solve. Roche paid $190M upfront for a Phase 1 asset aimed at it.
Wandering tumour cells go quiet, switch off the distress signals that would summon immune cells, and wait. Disrupt the silence and the immune system finds them again.
Compounding law lets a pharmacist tailor a medicine for one patient. Marketing an untested formulation to clinics is a different activity wearing the same name.
Every previous narcolepsy drug fought the symptoms. This one replaces the signal the missing neurons stopped sending.