Condensate Therapeutics for Targeted Diseases
In addition to physiological functions, biomolecular condensates are associated with disease pathologies such as neurodegenerative diseases, cancer, viral infections, and other diseases. In diseases pathological condensates may form ab initio or normal physiological condensates may be dysregulated or lost. Because of the broad biological effects of biomolecular condensates, they have emerged as potential therapeutic targets for these diseases. Many proteins and nucleic acids of high therapeutic importance, including many targets previously considered "non-druggable", act in condensates. In addition, there is new evidence that condensates are "druggable", for example:
- Some approved drugs have been shown to break down into condensates.
- High-inclusion cell screens have identified drug-like molecules that regulate the condensate behavior in a selective manner.
- Post-translational modifications can strongly regulate condensate formation, behavior, and solubilization.
Fig. 1. Therapeutic condensates in different diseases. (Niu X, et al, 2023)
Our Solutions
CD BioSciences offers a comprehensive biomolecular condensate pathology functional analysis service to analyze liquid-liquid phase separation (LLPS) and biomolecular condensates in a variety of diseases with the goal of developing therapeutic targets. Based on our condensate-based drug discovery platform, CD BioSciences develops innovative condensate therapeutics for difficult-to-treat diseases, including:
In addition, we are committed to developing innovative treatments for metabolic diseases such as insulin resistance and diabetic complications.
We offer a multidisciplinary approach to identify and localize specific condensates associated with specific diseases. By understanding the molecular syntax and unique characteristics of condensates, our experts can design condensate therapy that selectively interact with targeted condensates, which can disrupt these pathological condensates and prevent the accumulation of toxic protein aggregates, thereby producing the desired therapeutic outcome.
- Direct and Indirect Modulation Strategies
Direct condensate therapy interacts with the components of the condensate itself, influencing their behavior and disrupting the function of the condensate.
Indirect condensate therapy, on the other hand, targets upstream regulators of condensate formation or maintenance, including modulating the activity of enzymes, molecular chaperones, or other regulatory factors involved in condensate kinetics. This approach allows for intervention at multiple steps within the signaling cascade, offering a broader range of therapeutic possibilities.
- Phenotypic Readout and Drug Screening Strategies
To identify effective condensate therapies, CD BioSciences utilizes a range of phenotypic readouts and advanced screening methods. Our high-throughput screening (HTS) assays combine a variety of readout techniques, including traditional fluorescence microscopy and emerging technologies. These methods allow us to assess the impact of condensate therapy on condensate formation, composition, and function.
- Machine Learning (ML) Strategies
By analyzing complex data sets and considering the observed condensate phenotypes, composition, and possible confounding in screening hits, we can optimize the discovery of effective and selective condensate therapies.
Advantages of Our Condensate Therapeutics for Targeted Diseases
- By targeting the dysregulated condensate behind a specific disease, we can achieve greater specificity and minimize off-target effects.
- The dynamic nature of condensates allows for adaptability and flexibility of therapeutic strategies, making them suitable for personalized medical approaches.
- Our condensate therapeutics can be used in combination with existing therapies to enhance efficacy or overcome drug resistance.
CD BioSciences utilizes state-of-the-art methods to develop novel condensate therapeutics for targeted diseases. By harnessing the power of condensates, our goal is to revolutionize the therapeutic field and bring hope to patients suffering from a wide range of diseases. If you are interested in our solutions, please contact us now.
Reference
- Niu X, Zhang L, Wu Y, et al. (2023) Biomolecular condensates: Formation mechanisms, biological functions, and therapeutic targets[J]. MedComm, 4(2): e223.
For research use only, not intended for any clinical use.
Related Services
