Vybion Drug Pipeline

INT41 has received Orphan Drug Designation from the FDA for Huntington’s disease. IND enabling studies for INT41 Gene Therapy for Huntington’s disease are in place following a preIND meeting with the FDA in in February 2022 in response to the briefing book prepared by our consultants at Latham BioPharm, a world class consulting group based in Cambridge MA.

  • 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 age onset inversely correlated to polyQ length. The Huntingtin protein is critical for vesicle transport, cell-cell communication and cell division and is required for neuron development.

INT41 cell-based and animal model studies:

  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. INT41 may be a useful therapeutic in several of these diseases.

Patents for INT41 have been issued in North America, Asia, Australia, India and Europe.