Valbenazine Approved, HD History, Gene therapy success

FDA approves Valbenazine to treat HD chorea
Last week, on August 18th, Neurocrine Biosciences shared that the FDA has approved their drug valbenazine, also known as INGREZZA, to treat chorea in people with HD. While valbenazine doesn’t slow or stop HD, it may be helpful for managing involuntary movements that may interfere with day-to-day activities or safety for people with HD. It has been approved for several years to treat another disorder involving unwanted movements, tardive dyskinesia. HDBuzz broke down the specifics of how the drug works and its history in their most recent article; read the details here.

This Month in HD History 
The exact sequence and location of the gene that causes Huntington’s disease was published in 1993, but the hunt to find it had been on for some time beforehand. An important step towards identifying the gene’s exact location was first narrowing down its neighborhood. In August of 1987, HD researchers led by Dr. James Gusella reached this major milestone toward locating the huntingtin gene. The researchers reported that the huntingtin gene lies at the tip of the 4th chromosome—one of the 23 packages of genetic information we inherit from each of our parents. This was a monumental step towards identifying the huntingtin gene, and Dr. Gusella has continued to make major contributions to HD genetics research since.  

Gene therapy sees early success in treatment of Rett Syndrome 
Last week, Taysha Gene Therapies announced that they are moving forward with their Ph I/II study of TSHA-102 for treatment of Rett Syndrome based on positive results in the first person to be dosed. This company is focused on developing treatments for genetic brain disorders that result from mutations in a single gene. TSHA-102 is an experimental genetic drug designed for people with Rett syndrome, a rare disease that causes challenges with speech, hand-use, and coordination. Four weeks after dosing, a study participant who received the drug has begun to show improvements in their disease symptoms, and the safety profile of the drug appears to be strong. While TSHA-102 is not for people with HD, it is exciting to see positive news for gene therapies in other brain disorders involving one gene, and it is cause for hope that HD trials will see similar successes in the future. You can read more about this news and TSHA-102 here.