Mitochondrial Discovery Program
Current Knopp Research
Confronting New Challenges
The potential of dexpramipexole to improve bioenergetic efficiency creates exciting discovery opportunities. To pursue these opportunities, Knopp established its mitochondrial discovery platform.
With the goal of ultimately developing next-generation mitochondrial mediators, Knopp scientists leverage the pharmacology of dexpramipexole as a starting place for discovery. They confront the challenge from both a systems level and a reductionist approach, using each perspective to inform the other.
Knopp’s discovery scientists have extended earlier observations that dexpramipexole protects stressed neurons and other cell types from dying in preclinical models. Their laboratory research includes initial evidence of neuroprotection by dexpramipexole in multiple in vitro models of cell injury death.
Seeking the Breakthrough Treatments of Tomorrow
Knopp has initiated a medicinal chemistry program directed to the discovery of novel mitochondrial mediators. In this effort, our medicinal chemists begin with a major advantage.
Their advantage is the use of dexpramipexole as a starting point for a new generation of mitochondrial mediators. Dexpramipexole exhibits numerous advantageous physico-chemical properties, including its high water solubility, its ability to penetrate the blood-brain barrier, and its absence of metabolism by the liver. By associating the structural elements of the dexpramipexole molecule with its activity in cells, Knopp’s medicinal chemists can determine many of the properties for designing new molecules to enhance mitochondrial function.
In this effort, Knopp’s chemists work closely with their colleagues in biology. While the pharmaceutical industry grows increasingly specialized, with diverse functions often located on different continents through outsourcing, Knopp’s chemists and biologists work side-by-side, to accelerate the iterative process of discovering new molecules that may become the breakthrough treatments of tomorrow.
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.