Targeting the Lungs to Breathe Easier
4D-710 is being evaluated in the currently-enrolling AEROW clinical trial, a 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 endpoints of the study are safety, tolerability, and definition of the maximum-tolerated dose and/or Phase 2 dose level. Secondary endpoints include assessments of clinical activity, including quality-of-life and lung function, plus exploratory endpoints on the feasibility of detecting transgene transfer and CFTR transgene expression as measured in bronchoscopic biopsies and brushings.
The mission of the Cystic Fibrosis Foundation is to cure cystic fibrosis and to provide all people with cystic fibrosis the opportunity to lead long, fulfilling lives by funding research and drug development, partnering with the cystic fibrosis community, and advancing high-quality, specialized care.
Language courtesy of Cystic Fibrosis Foundation
Emily’s Entourage is an innovative 501(c)3 foundation that accelerates research for individuals in the final 10% of the CF population that do not benefit from existing mutation-targeted therapies, including those with nonsense mutations of CF. Since 2011, Emily’s Entourage has awarded millions of dollars in research grants, launched a now-acquired CF gene therapy company, developed a patient registry and clinical trial matchmaking program to accelerate clinical trial recruitment, and led worldwide efforts to drive high-impact research and drug development.
Language courtesy of Emily’s Entourage