Turning off the Huntington's gene
On 11 December 2017, Ionis Pharmaceuticals announced successful results from a Phase I/IIa trial of their drug IONIS-HTTRx, which is injected through the spinal canal and targets the underlying cause of Huntington’s disease (HD). IONIS-HTTRx, an antisense oligonucleotide (ASO), interferes with the translation of genetic information (mRNA) to the disease-causing protein: huntingtin (htt).
According to Ionis, the treatments were safe and tolerable, and caused a measurable dose-dependent reduction in the concentrations of htt within cerebrospinal fluid (CSF). The results made the headlines, and Roche has exercised an option to license the treatment for further development. This is very promising news. But I must caution that this is just one step in a chain of clinical-trial testing; the drug has not yet shown (nor has it had a fair chance to show) that it is an effective clinical treatment for HD.
Read More: http://bit.ly/HuntingtonGene
According to Ionis, the treatments were safe and tolerable, and caused a measurable dose-dependent reduction in the concentrations of htt within cerebrospinal fluid (CSF). The results made the headlines, and Roche has exercised an option to license the treatment for further development. This is very promising news. But I must caution that this is just one step in a chain of clinical-trial testing; the drug has not yet shown (nor has it had a fair chance to show) that it is an effective clinical treatment for HD.
Read More: http://bit.ly/HuntingtonGene
