About Cystic Fibrosis
Cystic Fibrosis is the most fatal inherited disease, affecting more than 70,000 patients worldwide. Cystic Fibrosis causes impaired lung function, inflammation and bronchiectasis and is commonly associated with repeat and persistent lung infections due to the inability to clear thickened mucus from the lung, often resulting in frequent exacerbations, hospitalizations and eventual end-stage respiratory failure.
4DMT Pipeline Product: 4D-710
Our lead product candidate in the lung therapeutic area, 4D-710, is wholly-owned and under development for the aerosol treatment of Cystic Fibrosis lung disease. We produced 4D-710 using a proprietary vector designed for efficient aerosol delivery to the lung airway and to have enhanced penetration through mucus, efficient airway cell transduction and transgene expression, and resistance to pre-existing antibodies in the patient population.
While several products have been approved for the treatment of Cystic Fibrosis, they fall short of restoring normal lung function in most patients, and these chronic therapies require daily dosing for the patient’s lifetime. Additionally, approximately 10% of patients remain without a corrective treatment option.
We expect to initiate IND-enabling study activities with 4D-710 in the first half of 2020.