One failed Phase 3 trial erased roughly 44% of Ultragenyx's share price in a single session. But the company did not wake up on September 3 as an empty biotech shell. It still has a growing commercial business, a newly approved gene therapy, and a second gene-therapy FDA decision scheduled for September 19. The question is whether the market has correctly repriced a damaged platform—or thrown away too much with the failed Angelman program.

Five drugs are approved for bladder cancer that stops responding to BCG, and every one of them requires carcinoma in situ. For papillary-only disease there is nothing. ImmunityBio asks the FDA to change that on 6 January 2027.

Acute myeloid leukemia in second remission is one of the few settings in oncology with no approved treatment at all. What that means, which medicines patients receive instead, and where galinpepimut-S would fit if the phase 3 succeeds.