Tominersen, formerly known as IONIS-HTTRx and RG6042, is an investigational antisense medicine designed to target the underlying cause of Huntington’s disease (HD) by reducing the production of all forms of the huntingtin protein (HTT), including its mutated variant (mHTT).
About Huntington’s Disease
HD is an inherited genetic brain disorder that results in the progressive loss of both mental faculties and physical control. It is caused by the expansion of the cytosine-adenine-guanine (CAG) trinucleotide sequence in the HTT gene. The resulting mutant HTT protein is toxic and gradually destroys neurons. Symptoms usually appear between the ages of 30 and 50 and worsen over a 10 to 25-year period. Ultimately, the weakened individual succumbs to pneumonia, heart failure or other complications. Presently, there is no effective treatment or cure for the disease, and currently available medicines only mask the patient’s symptoms but do not slow down the underlying loss of neurons.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.