STAT+: RA Capital backs upstart rival to Ultragenyx and Ionis in rare brain disease — SkimNews
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- RA Capital and a group of other investors committed $175 million to fund Oak Hill Bio, a startup built around an experimental Angelman syndrome therapy
- Oak Hill Bio is reviving a drug that Roche had previously shelved, betting the discarded molecule can outperform rivals in a rare neurodevelopmental disease
- Ultragenyx Pharmaceutical and Ionis Pharmaceuticals are both already running Phase 3 trials of similar Angelman syndrome drugs, putting Oak Hill behind on the development timeline
- Ultragenyx is set to report Phase 3 results this year, giving Oak Hill a near-term competitive benchmark to measure its molecule against
- Oak Hill Bio is making a best-in-class bet rather than a first-to-market play, wagering its molecule is superior to the two Phase 3 contenders
Why it matters: Oak Hill is entering an Angelman syndrome race where two competitors are already in Phase 3 and Ultragenyx will deliver a pivotal readout this year. A $175 million round from RA Capital signals serious investor conviction that a Roche castoff can leapfrog later-stage rivals on efficacy or safety, not speed.
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