12. A New Treatment For Huntington’s Disease
What It Can Do: Blocks The Huntingtin (HTT) Gene From Creating Toxic Proteins
Discovered By: Ionis Pharmaceuticals
Est. Cost: Unknown*
Huntington’s disease is a congenital degenerative disease that affects the brain cells of 1 out of every 10,000 people (about 750,000 people worldwide). Recently, a treatment has been trialed that is focused on aiding those in the early signs of the disease by using antisense oligonucleotide to block the huntingtin gene from creating the toxic depilating proteins.
A New Treatment For Huntington’s Disease ©Kateryna Kon / Shutterstock.com
In the spring of 2021, after not showing results that were indicative of keeping the program running, the trial concluded earlier than expected. Many lessons were, however, taken away from this trial that will be used to, hopefully, better direct future studies.