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Long-term follow-up of AAV8-mediated OTC gene therapy in adults with late-onset OTC deficiency
2022-501146-30-00Phase 2Authorised
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Nov 2022
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 5
01
Overview
Phase 2, open-label, single-arm trial of avalotcagene ontaparvovec in ornithine transcarbamylase deficiency, sponsored by Ultragenyx Pharmaceutical Inc. Planned start Nov 2022; status: authorised.
02
Drugs
avalotcagene ontaparvovec
03
Conditions
ornithine transcarbamylase deficiency
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