Clinical stage developer of Gene Therapies for neurodegenerative diseases with a new drug class asset and a platform for further discovery. Vybion has developed an Intrabody (intracellular antibody scFv fragment), called INT41, for Huntington's disease that when delivered by rAAV in an animal model delays motor function and cognitive loss. The mechanism of action is the elimination of the N-terminal toxic fragment that drives gene dysregulation and neuron loss. The Company has developed the IND enabling program following discussions with the FDA and has received Orphan Drug Designation for the FDA as well. INT41 is also the lead candidate for Gene Therapy in spinal muscular atrophy (SMA) and spinal cerebellar ataxia (SCA) 1, 3 and 7. Additional neurodegenerative disease development with similar accumulation protein fragments due to a breakdown in proteostasis include amyotropic lateral sclerosis (ALS) and frontal temporal dementia (FTD). The company operates virtually.
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