Vybion,

Vybion developed a novel vectorized intrabody (INT41) for Huntington’s disease, a devastating inherited orphan disease that results in the progressive loss of both motor and cognitive function. INT41 is effective in both cell-based system and animal models. INT41 has Orphan Disease Designation from the FDA and a pre-IND meeting defined the basic elements of the IND enabling package. INT41 binds to an epitope that is unavailable in the functional molecule, but appears in the toxic degradation fragment that accumulates in the nucleus, binds to DNA via the polyglutamine and alters gene expression detectable years before the onset of symptoms. INT41 stabilizes this fragment and facilitates its degradation. In addition to Huntington’s disease, Vybion is testing INT41 as a therapy for spinobulbar muscular atrophy (SBMA) and spinocerebellar Ataxia 1, 3 and 7 (SCA), two closely related Orphan diseases with an identical target sequence and polyglutamine expansion as seen in Huntington’s disease.