P-OTC-101

P-OTC-101 is a liver-directed gene therapy developed for the treatment of urea cycle disease caused by congenital mutations in the ornithine transcarbamylase (OTC) gene, specifically targeting ornithine transcarbamylase deficiency. The therapy utilizes a hybrid delivery system combining adeno-associated virus (AAV) vectors and lipid nanoparticles to deliver genetic material encoding the functional OTC gene directly to liver cells. This approach aims to restore normal enzyme function in patients with OTC deficiency, an often fatal or severely debilitating metabolic disorder characterized by impaired ammonia detoxification. The program is being developed by Poseida Therapeutics and remains in preclinical development[1][2][3][6][9][10].