Vybion, Inc. (“Vybion†or “the Companyâ€) is a development stage company with a patented technology platform used to develop a novel therapy (INT41) for Huntington’s disease, a devastating inherited orphan disease that results in the progressive loss of both motor and cognitive function. INT41 is a small antibody fragment (Intrabody) that is effective in both cell-based system and animal models. Recent mechanistic data reveal the ability of INT41 to inhibit disease pathology in both cell-based systems and animal models to specific biochemical events by neutralizing toxic Huntingtin mutant protein fragments in the nucleus. INT41 binds to an epitope that is not available on the whole molecule as determined by immunoprecipitation and affinity chromatography.
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.
The linkage of the action of small antibody fragments to inhibition of specific cellular pathology in polyglutamine based disease, is a clear step forward in establishing and extending the antibody/Intabody value proposition to this new drug class.