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DTX501
- Development stage
- Preclinical
- Lead developer
- Ultragenyx Pharmaceutical
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
DTX501 is a gene therapy product using the AAV8 vector for the treatment of **Phenylketonuria (PKU)**. It is developed by Dimension Therapeutics and targets the liver to deliver the PAH gene, which is deficient in PKU patients.
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