Ultragenyx Announces Additional Positive Multi-Year Durability Data from Phase 1/2 AAV Gene Therapy Studies
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Monday, November 29, 2021
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Across all patients in the Phase 1/2 study, to date there have been no infusion-related adverse events, no treatment-related serious adverse events, and no dose-limiting toxicities reported.
Key Points:
- Across all patients in the Phase 1/2 study, to date there have been no infusion-related adverse events, no treatment-related serious adverse events, and no dose-limiting toxicities reported.
- It is caused by a defective gene coding for the enzyme G6Pase-, resulting in the inability to regulate blood sugar (glucose).
- DTX401 is an investigational adeno-associated virus (AAV) type 8 gene therapy designed to deliver stable expression and activity of G6Pase- under control of the native promoter.
- DTX301 is an investigational AAV type 8 gene therapy designed to deliver stable expression and activity of OTC following a single intravenous infusion.