STAT+: RA Capital backs upstart rival to Ultragenyx and Ionis in rare brain disease

RA Capital and a group of investors have provided $175 million in funding to launch Oak Hill Bio, a startup aiming to develop an experimental treatment for Angelman syndrome. The newly backed company plans to revive a drug previously shelved by Roche.

With this funding, Oak Hill Bio enters a competitive rare disease space. The startup will rival Ultragenyx Pharmaceutical and Ionis Pharmaceuticals, both of which are already conducting clinical development for potential therapies targeting the same rare brain condition.