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Science

Condensates: Cell biology 2.0

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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 a condensatopathy.
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

Key publications

ERSA powered by ERSAi™: our proprietary AI/ML-powered platform for target identification and c-mod discovery & development

We combine world-class drug discovery experience, state-of-the-art infrastructure, deep condensate biology expertise, proprietary AI/ML tools, and openness to do things differently to translate new science of condensates into medicines  for diseases of high unmet need.

To this end, we built ERSA – our flexible integrated platform. Powered by our AI/ML-platform ERSAi™, ERSA supports the four pillars of our innovative approach to R&D: (1) target discovery, (2) condensate modulating drug (c-mod) discovery, (3) c-mod optimization and (4) c-mod development

ERSA, fully integrated with ERSAi, is supported by an in-house managed, proprietary chemical library, and is built with flexibility in mind, to drive a wide range of discovery and development efforts for any disease area of choice.

Take a virtual tour of our state-of-the-art facilities in Boston to learn more.

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Patents

May 2018

System and Method for Classifying and Segmenting Microscopy Images with Deep Multiple Instance Learning” (U.S. Patent No. 20180137338) covering novel technology for microscopy image analysis using machine learning.

The issued patent claims a neural network architecture and methods to classify microscopy images of cells.

May 2022

Methods of Screening for Condensate-Associated Specificity and Uses Thereof” (U.S. Patent No. 11340231) covering novel technology for screening compounds capable of modulating biomolecular condensates with high selectivity.

The issued patent claims key methods to identify potential drugs that can modulate particular biomolecular condensate components with precision, which can lead to the development of novel targeted therapies to treat cancer, neurodegenerative, cardiovascular, metabolic, infectious and many other diseases.

November 2022

Methods of Characterizing Condensate-Associated Characteristics of Compounds and Uses Thereof” (U.S. Patent No. 11493519) covering novel technology for partitioning and identifying compounds which preferentially partition into condensates of interest.

The issued patent is the first patented technology for measuring how a compound is entering or partitioning into a target condensate. This breakthrough technology will direct a compound to selectively target a single molecule in a condensate to alter disease.

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Scientific animation: Visual Science