Vybion Drug Pipeline

Vybion Drug Pipeline

Huntington's Disease:

  • Huntington's disease affects 5-7 individuals per 100,000 population globally
  • Progressively lethal neurodegenerative disease
  • $2.5B annual cost to healthcare
  • In US 30,000 patients and 150,000 at risk

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)

  1. Reduced loss motor function and had dramatic effects on cognitive function in the R6/2 animal model
  2. Increased turnover of mutant huntingtin "toxic" degradation fragments
  3. Reduced Gene Dysregulation
  4. Blocks binding of "toxic" mutant huntingtin fragments to chromatin/DNA
  5. Binds to huntingtin degradation fragments and does not bind to full length huntingtin protein

Polyglutamine Diseases:

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.