Chemical Biology and Therapeutics Science
Mission

The primary mission of the Chemical Biology and Therapeutics Science (CBTS) Program at the Broad Institute is to support community engagement in innovative chemical biology and therapeutics research.

X-ray co-crystal structure of B52 compound (cyan) binding and inhibiting Cyclophilin D protein
Credit: Aziz Rangwala
X-ray co-crystal structure of B52 compound (cyan) binding and inhibiting Cyclophilin D protein

About

The Chemical Biology and Therapeutics Science (CBTS) Program at the Broad Institute brings together a talented and diverse group of researchers that share a deep commitment to advancing biology and medicine through the use of chemistry and related molecular approaches. Recent advances have expanded the scope of chemical biology and therapeutics science to include: 

  • Breakthroughs in the use of macromolecules to study biology and modify our genomes and proteomes in therapeutically relevant ways.
  • Development of technologies that have transformed how small-molecule and macromolecular structure space can be explored to rapidly identify molecules with remarkable biological properties.
  • Innovative use of chemistry to provide an unprecedented view of previously unseen processes in living systems.
  • Integration of data from multiple technologies and knowledge sources to better understand the roles of small molecules in living systems. 
     

Chemical Biology and Therapeutics Science

Areas of Focus

Recent publications

Other Chemical Biology and Therapeutics Science Program publications
UM171 glues asymmetric CRL3-HDAC1/2 assembly to degrade CoREST corepressors.
CTCF/RAD21 organize the ground state of chromatin-nuclear speckle association.
Building the first base editors.
base editing of Angptl3 via lipid nanoparticles to treat cardiovascular disease.
Oxidation of retromer complex controls mitochondrial translation.
Covalent adduct Grob fragmentation underlies LSD1 demethylase-specific inhibitor mechanism of action and resistance.
Programmable gene insertion in human cells with a laboratory-evolved CRISPR-associated transposase.
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.
In vivo base editing extends lifespan of a humanized mouse model of prion disease.
Systematic empirical evaluation of individual base editing targets: validating therapeutic targets in USH2A and comparison of methods.

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