Ultragenyx Pharmaceuticals’ apazunersen did not meet the goals of its Phase 3 test in Angelman syndrome, a rare genetic neurological disorder with no FDA-approved therapies. The disappointing result has some readthrough to Ionis Pharmaceuticals and Oak Hill Bio, each in clinical development with Angelman drugs designed to work in a similar way as Ultragenyx’s failed molecule.
Scribe Therapeutics’ IPO will support a pipeline led by a genetic medicine that represses expression of its target gene without making permanent changes. Common chronic cardiometabolic conditions are Scribe’s initial focus as the biotech works to bring genetic medicines beyond rare diseases.
In an interview, Kyan Health Co-Founder and Chief Commercial Officer Konstantin Struck discussed how Kyan gives mid-market and enterprise employers access to premium workforce mental healthcare, at a price point that is affordable.