SKY-0515 for treating Huntington’s disease

SKY-0515 is currently being developed for the treatment of Huntington’s disease. Huntington’s disease is a genetic brain disorder that slowly affects how a person moves, thinks and behaves. It is a progressive and ultimately fatal disease that usually starts in adulthood and gets worse over time. Huntington’s disease is caused by a faulty gene, the Huntingtin gene (HTT), being passed onto a child by one of their parents. Each child of a Huntington’s disease patient has a 50% chance of inheriting the disease. Symptoms of Huntington’s disease usually start in adults aged between 40 and 50, but they can happen at any age. Early symptoms can include difficulty concentrating and planning tasks, memory problems, low mood, depression and anxiety, changes in behaviour and small twitching or jerking movements (chorea). Later symptoms can include difficulty swallowing and speaking, losing weight, challenges with movement and more severe mood or personality changes, and a total loss of independence and eventually premature death. Current treatment options for patients with Huntington’s disease are limited to managing symptoms, so there is a need for additional treatment options that targets the underlying cause of the disease.