Tag: #raredisease

Neurocrine to Buy Soleno for $2.9 Billion, Picks Up Prader‑Willi Drug VYKAT XR

Neurocrine will acquire Soleno for $2.9B to add VYKAT XR, the newly approved Prader‑Willi hyperphagia drug, aiming to expand its rare‑disease portfolio.

#biotech, #m&a, #raredisease

FDA proposes new pathway to approve one-patient gene-editing drugs for ultra-rare diseases

New FDA draft guidance would let individualized CRISPR and RNA therapies win approval with tiny studies, using strong biology and natural-history data.

#fda, #geneediting, #raredisease, #crispr, #drugapproval