The Food and Drug Administration (FDA) has granted Orphan Drug designation to INT41 (Vybion), an investigational treatment for Huntington disease.

Treatment with INT41 involves the delivery of intrabodies (intracellular antibody fragments) through the use of a recombinant adeno-associated virus (AAV). In animal studies, the treatment was associated with improvement in both motor and cognitive function.

Although still in its early stages, the Company believes INT41 has great potential for treating Huntington disease as well as other polyglutamine diseases (ie, spinobulbar muscular atrophy, spinocerebellar ataxia). These disease are characterized by progressive degeneration that causes impaired coordination. 

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“We are pleased to receive Orphan Drug designation for INT41, which has shown the potential to address an area of high unmet medical need,” said Lee Henderson PhD, CEO of Vybion.

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This article originally appeared on MPR