SERCA2b Small Molecule Program

Another focus of our research relates to a different from the of the SERCA enzyme, SERCA2b. Specifically these SERCA2b enzymes control calcium movement in the endoplasmic reticulum (ER) in all human cells. SERCA2b enzyme levels become deficient when cells are stressed and accumulate unfolded proteins in the ER, known as ER stress. There has been a proliferation of publications in the scientific and medical literature supporting the important role of ER stress in many diseases and conditions, including heart failure, diabetes and neurodegenerative diseases.

Using its proprietary, patented SERCA2 screening assay, Celladon has developed a broad platform of novel, first-in-class, small molecule modulators of the SERCA2b enzyme, which can correct underlying calcium dysregulation and ER stress. These proprietary compounds have demonstrated activity in multiple preclinical models of diseases and conditions.