Science
Focused on diseases of cellular cholesterol trafficking
Targeting a novel mechanism of action with a new class of therapeutics
Disease biology
Understanding the underlying biology driving neurodegenerative disease
Cholesterol balance inside the cell is essential for normal function.
Across a range of diseases, defects in intracellular cholesterol transport cause cholesterol to accumulate within cellular compartments. At the same time, cholesterol becomes insufficient in the membranes and other cellular regions that depend on cholesterol for signaling and essential biological processes. This disruption in cellular cholesterol homeostasis leads to metabolic stress, progressive cellular dysfunction, and ultimately cell injury and death, driving clinical symptoms.
Drug platform
We've made cyclodextrin-based therapeutics possible
It has not been possible to overcome the cholesterol trafficking defects that drive disease with existing medicines or modalities.
Pioneering work from multiple Nobel-winning labs (Brown and Goldstein) recognized that derivatized cyclodextrins—cyclic oligosaccharides with a hydrophilic exterior and hydrophobic cavity—are uniquely suited to traffic accumulated intracellular cholesterol. Through our fully integrated discovery capabilities, Beren is advancing a growing pipeline of unique derivatized cyclodextrins that traffic accumulated cholesterol for cellular use or elimination, restoring cellular cholesterol homeostasis.
Our first program: restoring cholesterol trafficking in Niemann-Pick disease type C
Unbalanced cellular cholesterol plays a central role in Neimann-Pick disease type C (NPC). In NPC, defects in lysosomal cholesterol transport cause unesterified cholesterol to build up within the endolysosomal compartment.
