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.
The HAV is a bio-engineered vascular tube for dialysis access in patients with ESRD. It utilises human vascular cells to form a mechanically strong, engineered tube similar to native blood vessels. The HAV is among the first generation regenerative medicine aiming to generate artificial biological structures used to repair or replace damaged tissues and organs. If licensed the HAV will provide a treatment option for patients to use a prosthesis for vascular access in haemodialysis patients with ESRD.
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.