Condensates-Resident Small Molecules Therapeutic Solutions

Intrinsically disordered structural domains are considered non-druggable because they do not form stable binding pockets for small molecules and therefore have not been prioritized in drug discovery. However, such proteins have the potential to be targets in highly relevant disease areas and have the propensity to phase separate into biomolecular condensates. This phenomenon has naturally aroused great interest in the field of drug discovery. While the direct joining of condensate components may allow the expected drug to occupy the condensate, the drug may also be concentrated within the condensate due to the transient contact network without high affinity for the specific target. By interacting with the chemical environment of the condensate, small molecules can strongly influence the nature of the condensate and modulate condensate formation or dissolution. Therefore, the use of small molecule ligands to target condensates may be a promising therapeutic strategy.

Fig. 1. Regulatory methods of biomolecular condensates. (Niu X, et al, 2023)

Our Solutions

CD BioSciences offers professional condensate-resident small molecules therapeutic solutions to find small molecules that bind directly to the proteins that make up the biomolecular condensate itself. Our R&D team develops innovative approaches and technologies to identify and optimize small molecule lead compounds to address a variety of disease indications such as neurodegenerative diseases, cancer, cardiovascular diseases, and infectious diseases.

We provide cutting-edge solutions in the field of condensate-resident small molecules therapeutics. Our integrated platform covers a range of services, including:

• Target Identification and Validation
  We use state-of-the-art technologies, such as high-throughput screening and advanced imaging analysis, to identify key targets in biomolecular condensates. Through rigorous validation studies, we can confirm the functional relevance of these targets and their potential as points of therapeutic intervention.
• Small-Molecule Discovery and Optimization
  With our expertise in medicinal chemistry and drug discovery, we work to discover and optimize small-molecule lead compounds specifically tailored to target biomolecular condensates. Through a combination of computational modeling, virtual screening, and chemical synthesis, we strive to develop effective and selective small molecule candidates.
• Interaction Characterization
  Our multidisciplinary team employs a range of techniques, including biophysical assays, structural biology, and molecular dynamics simulations, to analyze the interactions between small molecules and condensates.
• Effectiveness and Safety Assessment
  We conduct rigorous preclinical studies and utilize cellular and animal models to evaluate the therapeutic potential, pharmacokinetic and toxicological profiles of our small-molecule drug candidates. This comprehensive evaluation allows us to identify lead compounds with optimal efficacy and safety for further development.
• Translational Research and Clinical Development
  In collaboration with leading academic institutions, research organizations, and pharmaceutical companies, CD BioSciences is committed to translating our condensate-resident small molecules therapeutics into clinical applications. Our goal is to bring innovative therapies targeting biomolecular condensates to patients in need.

Advantages of Our Condensates-Resident Small Molecules Therapeutic Solutions

• Small molecules are able to distribute between the condensate and the surrounding solution based on a partition coefficient. This distribution allows small molecules to influence the nature of the condensate and possibly modulate its formation or dissolution.
• Small molecules can alter the phase behavior of phase-separated proteins by directly interfering with the ability of pathological proteins to self-condense into a liquid phase or by interfering with their recruitment to biomolecular condensates.

CD BioSciences is committed to harnessing the potential of condensate-resident small molecule therapeutics to address unmet medical needs in a variety of disease areas. If you are interested in our solutions, please contact us now.