About Cystic Fibrosis
Cystic fibrosis is the most common fatal inherited disease in the United States and results from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. 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 and hospitalizations and eventual end-stage respiratory failure. According to the Cystic Fibrosis Foundation, more than 30,000 people in the United States and more than 70,000 people worldwide are living with cystic fibrosis, and approximately 1,000 new cases of cystic fibrosis are diagnosed in the United States each year.
4DMT Pipeline Product: 4D-710
Our pulmonology product candidate is comprised of our targeted and evolved vector, A101, and a codon-optimized microCFTR transgene.
4D-710 is enrolling patients in an ongoing Phase 1/2 dose-escalation and dose expansion clinical trial in patients with cystic fibrosis who are ineligible for CFTR modulator therapy or who have discontinued therapy due to adverse effects. The primary endpoint of the study is safety and tolerability. Secondary endpoints include assessments of clinical activity including lung function, plus exploratory endpoints on the feasibility of detecting transgene transfer and microCFTR expression as measured in bronchoscopic biopsies and brushings.