STAT+: RA Capital backs upstart rival to Ultragenyx and Ionis in rare brain disease
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- RA Capital and a fleet of other investors are placing $175 million behind Oak Hill Bio, a startup reviving an experimental Angelman syndrome drug that Roche shelved.
- Oak Hill Bio is entering a three-way race against Ultragenyx Pharmaceutical and Ionis Pharmaceuticals, both of which are already running Phase 3 trials of similar Angelman syndrome drugs.
- Ultragenyx is expected to report Phase 3 results this year, setting an imminent competitive benchmark against Oak Hill's program.
- Oak Hill Bio is staking the round on the claim that it has the "best molecule" among the competing Angelman syndrome programs.
- Roche is cited as the originator of the drug now being revived by Oak Hill, having previously discontinued development of the asset.
Why it matters: Oak Hill is wagering $175 million on a revived Roche asset that will be measured against Ultragenyx's Phase 3 readout expected this year — meaning investors are paying for a molecule whose clinical competitiveness could be publicly validated or undermined by a competitor's imminent data within months.
