C207

C207 is a second-generation bifunctional small molecule designed to provide precise, tunable control over recombinant adeno-associated virus (rAAV) transcription. Developed by researchers at the University of North Carolina at Chapel Hill, C207 acts as a chemical epigenetic modifier by linking the small molecule ligand AP1867 to the bromodomain inhibitor JQ1. In this bioorthogonal platform, C207 binds to a modified FK506 Binding Protein (FKBP-F36V) fused to a zinc finger protein, which is targeted to a compact DNA-binding array upstream of a transgene. This recruitment of endogenous BRD4 specifically to the viral episome enables high-magnitude, reversible transcriptional activation of the transgene payload. C207 has demonstrated favorable pharmacokinetics in mouse models and is intended for post-delivery dose modulation in gene therapy applications.