Novel Huntington’s Therapy to Enter Clinical Development
Ionis Pharmaceuticals, Inc. — formerly Isis Pharmaceuticals — announced the U.S. Food and Drug Administration (FDA) has approved its Orphan Drug Designation application for IONIS-HTTRx as a potential treatment of Huntington’s disease (HD). Ionis’ IONIS-HTTRx is a generation 2.0+ antisense drug developed to treat HD by reducing the production of all forms of the huntingtin…