Mitochondrial Discovery Platform
Discovering Next Generation Compounds
The mitochondrion is the powerhouse of the cell, providing energy to every cell type in the body. Brain cells rank among the most energy-demanding cells in the body. The disruption of cellular bioenergetic activity has emerged as a common factor in multiple pathways leading to nerve cell death in diseases such as ALS, Parkinson's and other neurodegenerative disorders.
Drugs with the potential to improve or restore mitochondrial function are highly promising as treatments for those diseases. Dexpramipexole, which is in late-stage clinical development for the treatment of ALS, has emerged as a potential modulator of mitochondrial bioenergetics in preclinical models. Scientists at Knopp are making progress in characterizing the drug's mechanism of action, and have developed screening assays with the goal of discovering next generation compounds and advancing the pharmacology of dexpramipexole.
Publications of interest include:
Knopp's pipeline consists of investigational drug products that have not been approved by the U.S. Food and Drug Administration. These investigational drug products are still undergoing clinical study to verify their safety and effectiveness.