avalotcagene ontaparvovec

Avalotcagene ontaparvovec (DTX301) is an investigational gene therapy designed to treat ornithine transcarbamylase (OTC) deficiency, a rare genetic disorder affecting ammonia metabolism. The therapy uses an adeno-associated virus serotype 8 (AAV8) vector to deliver a functional copy of the OTC gene via single intravenous infusion. This enables stable expression and activity of the OTC enzyme in patients, aiming to restore normal urea cycle function and reduce neurocognitive damage caused by hyperammonemia. The drug is currently being evaluated in Phase 3 clinical trials for its efficacy in reducing ammonia levels and decreasing reliance on ammonia scavenger medications and protein-restricted diets. Avalotcagene ontaparvovec has received Orphan Drug Designation in the United States, European Union, and United Kingdom, as well as Fast Track Designation in the US[6].

Other names

Recombinant adeno-associated virus serotype AAV8 vector encoding human ornithine transcarbamylaseAdeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase