Wave Life Sciences announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to the investigational drug WVE-120101 for the treatment of Huntington’s disease (HD).
Huntington’s disease is a hereditary neurodegenerative disease that causes the progressive breakdown of nerve cells in the brain. It is a fatal disorder with currently no available treatments to slow down the progression or reverse the course of the disease.
WVE-120101 is an allele-specific antisense therapy which targets rs362307, a Single Nucleotide Polymorphism (SNP) that is associated with the disease-causing mutation in the huntingtin (HTT) gene.
For more information visit Wavelifesciences.com.