Investigational Drug for Huntington's Disease Granted Orphan Drug Status
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.
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