Hyperoxaluria

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 a mutation of the AGXT-gene that leads to decrease in the amount or function of alanine-glyoxylate aminotransferase (to convert glyoxylate to amino acid glycine). As a result of impaired function, overproduction of oxalate subsequently leads to excessive levels of oxalate excreted as waste product in urine. Calcium oxalate is formed secondary to this oxalate overproduction, leading to deposits of kidney and bladder stones injuring other organs and tissues in the body. Using AMA001 we intend to restore the serine-pyruvate aminotransferase activity (to prevent excessive oxalic acid secretion) in liver cells of patients with PH1 and therefore curing the disease.