Pipeline

Hypoxemic Pulmonary Disorders

Our preclinical and preliminary clinical investigation of GBT440 demonstrated the compound¬ís ability to increase the oxygen affinity of hemoglobin. This mechanism has important therapeutic potential beyond SCD and, as such, we are also planning to develop GBT440 as a potential treatment for acute and chronic hypoxemic pulmonary disorders.  

We are initially focusing these efforts on indications with significant unmet need, including idiopathic pulmonary fibrosis (IPF) and acute respiratory distress syndrome (ARDS), in which hypoxia is believed to play a key role in disease pathogenesis and adverse patient outcomes. These are conditions in which the lungs cannot supply adequate oxygen to the blood. While supplemental oxygen therapy is a well-established lifesaving treatment in acute and chronic hypoxemic conditions, it is associated with a number of risks, including injury or infection as a result of intubation. This highlights a significant unmet medical need which we believe can be addressed with a drug that improves oxygen uptake and delivery without the risks associated with supplemental oxygen delivery.

Our research suggests that hemoglobin modifiers such as GBT440 have the potential to enable increased oxygen uptake in the lungs, resulting in improved oxygen delivery to tissues. To date, we have established proof of concept for GBT440 analogs in three different animal models of hypoxia.