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- Ultragenyx prices Sanfilippo gene therapy at $4 million as it gains a second FDA approval; Bristol Myers halts Orum cancer drugThe FDA approved Fayuvi, the first gene therapy for Sanfilippo syndrome Type A, with a list price of $3.95 million. This marks Ultragenyx's second gene‑therapy approval since August, earning sella…
- BMS drops Orum’s targeted degrader after Phase 1 resultsBMS announced it is ending development of ORM-6151, a degrader‑antibody conjugate acquired from Orum Therapeutics. The decision follows a Phase 1 trial in acute myeloid leukemia and myelodysplasti…