Vybion Drug Pipeline
Huntington's is an inherited progressive neurological disease that leads to loss of motor and cognitive functions, characterized by the death of specific nerve cells in the brain. Huntington's disease results from a mutation that expands the CAG nucleotide encoding glutamine (Q) in the Huntingtin gene of patients to a length beyond 36 repeats of Q which results in disease. Although the precise function of the Huntingtin protein is unclear, it is involved in vesicle transport.
Vybion Intrabodies (INT41)
Several inherited neurologic diseases are caused by polyQ expansion. Nine diseases have been identified: Huntington's disease (HD), spinobulbar muscular atrophy (SBMA/SMA), six types of spinocerebellar ataxia (SCA). All patients with polyQ diseases present with progressive degeneration of a population of neurons in the central nervous system that are involved in motor control.