Vutrisiran for treating hereditary transthyretin amyloidosis

Vutrisiran is in clinical development for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with polyneuropathy. hATTR amyloidosis is an inherited condition that is caused by mutations in a gene known as the transthyretin (TTR) gene. This causes the liver to produce an abnormal version of the TTR protein, which accumulates as deposits in the tissues of the body (amyloidosis). These accumulated deposits can disrupt the structure and damage the function of the affected tissues causing symptoms such as pain, loss of sensation and weakness in the hands, arms, legs or feet. The disease can also affect involuntary body functions such as blood pressure, heart rate, and digestion. The effects and complications of the condition can lead to death within 3 to 15 years of symptoms developing. Currently there are limited treatment options for this disease, and treatment involves regular intravenous infusions.