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Rugonersen · RO-7248824-000

Oligonucleotide (Antisense oligonucleotide)

Phase 3 Most advanced trial
1 Registered trials

What is Rugonersen?

Rugonersen is an antisense oligonucleotide that reduces expression of UBE3A-AS, an antisense transcript that silences the paternal UBE3A allele. By inhibiting this transcript, rugonersen allows expression of the paternal UBE3A gene, restoring ubiquitin ligase activity in Angelman syndrome.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as
RO-7248824-000
Drug type
Oligonucleotide (Antisense oligonucleotide)
Therapy area
Neurology
In trials of
MEDP
FDA status
Investigational, not approved in the US. Most advanced trial: Phase 3
UNII
F77TA4U3SP

🧪 Clinical trials

TrialPhaseConditionStatusSponsor
NCT07605429 Phase 3 Angelman Syndrome Recruiting MEDP

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Angelman Syndrome

Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z

This data is for informational purposes only, not investment advice. BioRadar does not provide buy/sell recommendations. Past performance does not guarantee future results. Always do your own due diligence.