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What is Eladocagene Exuparvovec used for?

This gene therapy delivers a working copy of a gene into brain cells to treat a rare inherited disease affecting the nervous system. It is given as a one-time treatment directly into the brain.

How does Eladocagene Exuparvovec work?

Delivers functional copies of a gene to target cells using a viral vector, restoring expression of the deficient protein and correcting the underlying genetic defect.

Is Eladocagene Exuparvovec an approved drug?

Yes, Eladocagene Exuparvovec is an approved drug. It has reached clinical phase 4. It is classified as a Gene.

{# References & Data Sources section for drug detail pages. Renders standard pharmacological database links plus the drug's data_sources field. #}

References & Data Sources

• ChEMBL — European Bioinformatics Institute (EBI). CHEMBL4298189. Open-access bioactivity database.

Data aggregated from publicly available pharmacological databases. Last updated 2026-02-27.