IONIS-HTTRx, is an anti-sense drug that acts as a gene silencer to inhibit the production of Huntingtin (HTT) protein in people with Huntington's disease.

‘A new drug IONIS-HTTRx was found to be well-tolerated without any dose-limiting side effects, in the experiments conducted on rodents and monkeys.’

"It is very exciting to have the possibility of a treatment that could alter the course of this devastating disease," said principal investigator Blair Leavitt from the University of British Columbia in Vancouver, Canada. 




The findings will be presented at the American Academy of Neurology's 68th Annual Meeting in Vancouver, Canada, starting from April 15. Huntington's disease is passed from parent to child through a mutation in the HTT gene.
The mutation results in the production of a disease-causing HTT protein. Each child has a 50/50 chance of inheriting the gene mutation. Everyone who inherits the mutated gene would eventually develop the disease.
In the study, IONIS-HTTRx was found to be well-tolerated without any dose-limiting side effects, in the experiments conducted on rodents and monkeys. The drug is now in the initial phase of its clinical trial.
Source-IANS