Ulefnersen

DB18677InvestigationalGene Therapies

Ulefnersen: Overview.

Mechanism: Pharmacologically active on RNA-binding protein FUS
Curator reviewed
Code names: ION-363

Resolves to

Compound

Targets

What you can answer from here — as of October 09, 2026

1Protein targetEach mapped to UniProt, with action and pharmacological action

Listed above

2Clinical trialsPhase, status and sponsor resolved per trial

3+ReferencesStructured and connected to the statements they support

Which drugs share a target with Ulefnersen, and which of those have an active Phase 3 trial?

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