Lumasiran is in clinical development for the treatment of primary hyperoxaluria type I (PH1). PH1 is a very rare disease caused by certain genetic mutations, in which excess oxalate production results in the deposition of oxalate crystals in the kidneys and urinary tract. This leads to stone formation and kidney failure with significant morbidity and mortality. Treatment options for PH1 include vitamin B6 which is known to reduce the body’s production of oxalate, dietary recommendations to prevent kidney stones and combined liver-kidney transplantation before or after development of end-stage kidney failure.
Lumasiran, which is administered as a subcutaneous injection, is designed to reduce the levels of an enzyme called glycolate oxidase produced by the liver. Oxalate production is therefore inhibited. By reducing oxalate production, lumasiran has the potential to prevent the actual disease process that develops in PH1. If licensed, lumasiran may provide the first pharmacological treatment option for patients with PH1 who do not have any approved treatment.
The Human acellular vessel (HAV) is in clinical development to provide vascular access for haemodialysis patients with end-stage renal disease (ESRD). ESRD is a long-term irreversible condition where the kidneys do not work as well as they should as a result of chronic kidney disease. There is no cure for kidney failure and patients with this condition requires haemodialysis or a renal transplant for survival. Haemodialysis works by taking blood from the body and cleaning it through a machine to remove the toxins. Blood is filtered before being returned to the body. Vascular access is a way to reach the blood during haemodialysis.