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

STAT PlusBiotech RA Capital backs upstart rival to Ultragenyx and Ionis in rare brain disease Merger will help Oak Hill Bio revive old Roche drug for Angelman syndrome Manage alerts for this article Email this article Share this article By Jason MastJuly 27, 2026 General Assignment Reporter Jason Mast[email protected]Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. RA Capital and a fleet of other investors are putting $175 million behind Oak Hill Bio, a startup trying to revive an experimental Angelman syndrome drug that Roche shelved. Oak Hill is competing with Ultragenyx Pharmaceutical and Ionis Pharmaceuticals, both of which are already running Phase 3 trials of similar drugs for the same disease. Ultragenyx will have results this year.Advertisement Oak Hill is betting it has the best molecule. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+. Already have an account? Log in Monthly $39 Totals $468 per year $39/month Get Started Totals $468 per year Starter $30 for 3 months, then $399/year $30 for 3 months Get Started Then $399/year Annual $399 Save 15% $399/year Get Started Save 15% 11+ Users Custom Savings start at 25%! Request A Quote Request A Quote Savings start at 25%! 2-10 Users $300 Annually per user $300/year Get Started $300 Annually per user View All Plans To read the rest of this story subscribe to STAT+. Subscribe Log In biotechnology, drug development, rare disease, STAT+ Submit a correction requestReprints Jason Mast General Assignment Reporter Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. Newsletter Understand how science, health policy, and medicine shape the world every day Recommended Biotech July 27, 2026 STAT Plus: Argenx to buy Forte Biosciences for $2.2B Health July 24, 2026 FDA advisory panel narrowly rejects compounding of one peptide, backs two others Advertisement The Readout July 24, 2026 STAT Plus: Fatal gene-editing trial in China raises ethics and oversight questions Biotech July 23, 2026 STAT Plus: In win for RFK Jr., FDA advisory panel narrowly votes to allow compounding of unapproved peptides The Readout July 23, 2026 FDA panel confronting peptide controversy Subscriber Picks