OT-44

About OT-44

OT-44 is being developed as an innovative enzyme replacement therapy (ERT) for the treatment of Propionic Acidemia.

Propionic acidemia (PA) is a rare disorder caused by a deficiency in a mitochondrial enzyme, propionyl-CoA carboxylase (PCC). The enzyme is involved in metabolic pathways that process certain amino acids and fats. In most cases of PA, a neonatal-onset is observed and it is characterized by poor feeding, vomiting and somnolence. Later on seizures, coma, lethargy and eventually death occur. Both neonatal and late onset PA will experience devastating manifestations that include growth impairment, intellectual disability, basal ganglia lesions, pancreatitis, and cardiomyopathy. There is no treatment for PCC, and prevention of primary manifestations by dietary management and supplements is sub optimal. Management of acutely metabolic decompensated patients, is a medical emergency that requires emergency treatment. For some patients, liver transplantation is necessitated.

In contrast to other biologics in our pipeline, treatment of PA requires delivery of the enzyme into its natural environment, namely the specific cellular organelle- the mitochondria. For this, OT is optimizing and perfecting novel state-of-the-art strategies for specific targeting of PCC to the mitochondria. Our strategy has proven successful in tissue culture, and studies in animal models of PA are underway.