Condensate biology sparked a paradigm shift starting ~15 years ago (watch “Anthony Hyman: How cells form membraneless compartments”). We now understand that compartmentalization of cellular components into membraneless bodies (biomolecular condensates) is critical for regulation in time and space of myriad biological processes across all walks of life (watch “Intro to condensates”). We also understand that defects in the regulation of condensates lead to disease. Condensates represent a new class of drug targets that can be leveraged to discover and develop novel therapeutics that address diseases of high unmet need (watch “Condensate modifying drugs: a revolution in drug discovery”).
Explore this page to familiarize yourself with the specialized nomenclature in the field, foundational concepts and key background publications.
The Animation Lab is a great resource for introductory materials into the physics and biology of biomolecular condensates.
What are biomolecular condensates?
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According to the traditional view, the cell is compartmentalized by membrane-bound organelles such as the nucleus, mitochondria, and endoplasmic reticulum.
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But we now understand that there are also many different membrane-less organelles – condensates, formed by a process called phase separation.
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Condensates form and dissolve dynamically.
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Their behavior is governed by multivalent interactions between proteins and nucleic acids. Condensates compartmentalize and concentrate molecules, enabling key biochemical processes.
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Aberrant condensates drive many diseases. When something goes awry with a condensate’s composition or behavior, we call it condensatopathy.
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Condensatopathies occur when biomolecules inappropriately form a compartment, fail to do so, or when proteins localize to the wrong condensate. The consequence can be a toxic gain of function and/or a loss of function.
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Condensate modifying drugs (c-mods) are uniquely designed to hit condensate targets.
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c-mods treat condensatopathies and prevent or reverse disease by:
- preventing harmful protein sequestration
- driving key components back into condensates
- restoring healthy condensate properties and function
