Novel Huntington's Treatment Wins Orphan Drug Status

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Novel Huntington's Treatment Wins Orphan Drug Status
Novel Huntington's Treatment Wins Orphan Drug Status

Ionis Pharmaceuticals has announced that the Food and Drug Administration has granted Orphan Drug designation to IONIS-HTT for the treatment of Huntington's disease.

IONIS-HTT is the first target-directed therapy in clinical development designed to treat the underlying cause of Huntington's disease, a rare, genetic neurological disorder.

The Gen. 2.0+ antisense drug directly targets the cause of Huntington's disease by reducing the production of all forms of the huntingtin (HTT) protein. This mechanism offers a unique approach for the treatment of the disease.

There is currently no effective disease modifying treatment, and current approaches only focus on managing some disease symptoms.

Reference

Ionis Pharmaceuticals press release

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