Therapeutic Strategy

Focus on Cryptochrome Modulation

Genome-wide association studies, transgenic knock-out research, in vitro cellular manipulations and other models of disease have shown that:

  • A deficiency of cryptochrome is associated with, or can lead to, a number of diseases
  • Increased levels of cryptochrome can be associated with health and protection from disease

The company has developed proprietary stabilizers of cryptochrome that can increase cryptochrome levels in a targeted and transient manner1.  This allows targeted modulation of the core-clock with the goal of addressing dysregulation, thereby removing an underlying driver of a particular circadian rhythm associated disease.

Synchronicity Pharma has used these cryptochrome stabilizers to demonstrate preclinical efficacy across a wide range of preclinical disease models.  Synchronicity has also taken their lead program, SHP-1705, into the clinic and for the first time shown the ability to modulate human core-clock related gene expression.

(Data from Synchronicity’s Phase 1 first-in-human trial, SHP-1705-0012)

Synchronicity is therefore in a unique position to translate the promise of circadian biology advances into transformative therapeutics.

 

 

References:

  1. Humphries et al. “Carbazole-containing amides and ureas: Discovery of cryptochrome modulators as antihyperglycemic agents” Bioorg. Med. Chem. Lett (2017) 17:31225
  2. Unpublished data from Study SHP-1705-001