Ionis will launch its first commercial product through a co-commercialization agreement with AstraZeneca early next year, in preparation for its first wholly-owned drug launches soon to follow.

ReNAgade Therapeutics came out of stealth mode with $300m to fund its proprietary technology for delivery of RNA medicines – including coding, editing and gene insertion modalities – beyond the liver.

The difficulty of finding and retaining patients with orphan diseases translates into higher marketing costs and adds to other restrictions that limit the attractiveness of some orphan drugs. Will the IRA exacerbate matters?

The mid-sized company is under siege from hedge fund Farallon and faces a fight over its R&D strategy to follow aging blockbuster Cabometyx.