CT-101

CT-101 is a code name associated with two distinct investigational small molecule drug candidates. The first is a Class 1 histone deacetylase (HDAC) inhibitor being developed for the treatment of sickle cell disease (SCD). It works by inducing fetal hemoglobin (HbF) expression through the activation of γ-globin gene transcription, which helps mitigate the symptoms of SCD. Preclinical studies in humanized mouse models have shown that it increases F-cells and HbF levels while improving splenic health. The second candidate, also designated CT101, is a novel kinase inhibitor originally developed by Creabilis SA (later acquired by Sienna Biopharmaceuticals) for the topical treatment of inflammatory skin diseases such as atopic dermatitis and psoriasis. This version was identified using a systems biology approach and utilizes Low Systemic Exposure (LSE) technology to minimize systemic side effects.