Current Pipeline
ASO

Oak Hill Bio • rugonersen

Oak Hill Bio is exploring an investigational Antisense Oligonucleotide (ASO) approach for paternal gene activation as a potential treatment for Angelman syndrome.

Discovery & DevPre-clinicalPhase 1Phase 2Phase 3To Patients

About the Program

A therapeutic approach that uses modified RNA or DNA molecules that bind to the RNA of the UBE3A-ATS (UBE3A antisense transcript).

Investigational Drug Name
rugonersen (RO7248824)
Company Website
Oak Hill Bio

Questions about Clinical Trials?

Schedule a private, 1:1 appointment with Niki Armstrong, a genetic counselor who specializes in Angelman syndrome.

Meet with a Genetic Counselor

Program News

Oak Hill Bio: A Message to the Angelman Syndrome Community

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Rugonersen Development Update: First Participant Dosed in the Phase 3 BEACON Trial

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Update on Rugonersen Development and Planned Phase 3 Study  (BEACON)  

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Oak Hill Bio Announces Publication of Rugonersen Phase 1 Study Results in Nature Medicine

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Oak Hill Bio to continue the development of rugonersen, an investigational ASO previously owned by Roche.

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An update on Roche’s trials of rugonersen (RO7248824) in Angelman syndrome

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