Roche ends research on rugonersen in Angelman syndrome

26 June 2023

Swiss pharma giant Roche (ROG: SIX) will not begin any new trials of rugonersen (RO7248824) in Angelman syndrome, a rare genetic condition that affects the nervous system and causes severe physical and learning disabilities.

FAST, the US Angelman syndrome patient advocacy organization, reported the news last week, explaining that Roche had made the decision after looking at Phase I study results.

The problem was due to a lower level of efficacy than the Basel-based firm had hoped for, rather than any safety concerns.

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