Huntington’s disease (HD) is an inherited, degenerative brain disorder named after physician George Huntington, who first described the illness in 1872. It is also sometimes referred to as Huntington’s chorea, from a Greek word meaning “dance”, referring to the characteristic movement disorder that is a part of the illness. In 1993, scientists identified the gene that causes HD, huntington, and found this gene has a polyglutamine (CAG) expansion.
About 30,000 people in North America have HD, and another 150,000 are considered “at risk” for inheriting the illness because they have (or had) a parent with HD. Most develop HD by middle adult life, but about 10% will have onset prior to age 20 (juvenile HD) and another 10% after age 55. Men and women are equally likely to inherit the gene and develop the illness. HD can affect people of all ethnic groups, but is more common among those of European descent. There are currently no FDA approved treatments for HD.
One of EnVivo’s Huntington’s Disease programs is based on developing inhibitors to histone deacetylase (HDAC). HDAC inhibitors are being widely studied for use as cancer treatments and recently have also shown promise in mouse and invertebrate models of HD. EnVivo’s second Huntington’s Disease program is based on a repurposed generic drug that also has neuroprotective properties in EnVivo’s transgenic Huntington’s disease Drosophila and mouse models.