Primary Hyperoxaluria type 1
AMA001 is a gene replacement therapy product for the treatment of primary hyperoxaluria type 1 (PH1), a rare genetic liver disease with high unmet clinical need. PH1 is caused by mutations in the AGXT gene that encodes alanine-glyoxylate aminotransferase (AGT) that converts glyoxylate to glycine in the liver. As a result of the impaired AGXT function oxalate is overproduced resulting in excessive excretion of oxalate in the urine, in the deposition of calcium oxalate crystals in organs and joints and in the accumulation of kidney and bladder stones, eventually leading to premature death of the patients. Using AMA001 we intend to restore the AGT activity in the liver of PH1 patients, thereby potentially curing patients from the disease.