Methods To Generate Constrained Fluorinated Macrocycles (UCLA Case No. 2023-139)

UCLA researchers from the Department of Chemistry and Biochemistry have developed a novel method to generate fluorinated polycycles, which may be used for downstream drug discovery and development.

BACKGROUND: A significant amount of research in the field of organic chemistry centers around generating increasingly complex molecules that cyclize, forming covalent bonds and rings. These molecules hold clinical significance in their potential to be ligands for protein surfaces involved in protein-protein interactions, allowing them to serve as molecular “glue”. Various methods have been developed to produce these molecules, including using reagents that can induce ring forming reactions or  using scaffolding molecules. However, these methods are limited by the nature of the molecule. For instance, the scaffolding methods are limited to hydrophobic residues. Thus, developing new methods to efficiently generate macrocycles may hold impactful clinical significance and become extremely valuable for drug discovery.

INNOVATION: UCLA researchers have generated a novel method to generate complex fluorinated macrocycles using octafluorocyclopentane (OFCP). This can be done in a single flask at room temperature and does not require catalysts or heavy metals. OFCP can be used in two ways: (1) by directly polycyclizing linear molecules or (2) by adding reactive molecules to intercept cyclic intermediates. The generated polycycles closely mimic protein surface loops. Furthermore, a subset of the generated polycycles shield the polar surfaces using hydrogen bonding, allowing them to be permeable enough to pass through cell membranes. Thus, this technology can be used to design macrocyclic petidomimetics or potential ligands involved in protein-protein interactions that are valuable for drug discovery.

POTENTIAL APPLICATIONS:

  • Generating cell-permeable macrocycles that can be used as drugs
  • Novel methodology to produce cyclic peptides and peptidomimetics

ADVANTAGES:

  • This method can be used to generate a diverse set of cyclic peptides, providing a large pool for drug discovery
  • Some of the generated molecules shield their polar surface area, allowing them to exhibit high permeability

DEVELOPMENT-TO-DATE: UCLA researchers have generated a method to rapidly create complex fluorinated macrocycles, a subset of which mimic protein-protein interactions and exhibit high permeability.

Related Papers (from the inventors only): Mendoza, A., Bernardino, S., Dweck, M., Valencia, I., Evans, D., Tian, H., Lee, W., Li, Y., Houk, K., & Harran, P. (2023). Cascade synthesis of fluorinated spiroheterocyclic scaffolding for peptidic macrobicycles. Journal of the American Chemical Society, 145(29), 15888–15895. https://doi.org/10.1021/jacs.3c03071

KEYWORDS: Macrocycles, drug discovery, polycyclization, protein-protein interaction, loop structures, cell permeability, polysubstitution, PAMPA, peptides

Patent Information: