ssAAV8-OTC

**ssAAV8-OTC** is an investigational **AAV8-based in vivo gene therapy** designed to deliver a functional human **ornithine transcarbamylase** gene to hepatocytes for treatment of **ornithine transcarbamylase deficiency**, a urea-cycle disorder characterized by impaired ammonia detoxification. The product appears to be a **single-stranded adeno-associated virus serotype 8 vector** carrying an OTC transgene intended to restore hepatic OTC enzyme activity and thereby reduce hyperammonemia and related metabolic decompensation. Publicly visible evidence for this exact name is limited and mainly preclinical, including Selecta Biosciences materials describing **ssAAV8-OTC** used with ImmTOR nanoparticles to improve transgene expression, blunt anti-AAV humoral responses, and enable potential redosing; this exact construct name should be distinguished from the clinically advanced **DTX301 scAAV8OTC avalotcagene ontaparvovec**, which is a separate specific OTC gene therapy nomenclature used in human trials.

Other names

single-stranded AAV8-OTCAAV8-OTCAAV-8-OTCAAV 8-OTC