4D: Rare Pediatric Disease Designation for Cystic Fibrosis Treatment

 

• 4D-710 has demonstrated promising, reproducible, CFTR expression significantly above normal levels for seven patients across Cohorts 1 & 2 (1E15-2E15 vg) and durable improvement or stabilization of quality of life & pulmonary function for three patients through 12 months in Cohort 1
• Given above normal CFTR transgene expression (~400% of normal lung levels), dose exploration continues with evaluation of lower doses (Cohort 3 dose 5E14 vg)
• Interim data update from Phase 1/2 AEROW clinical trial expected in mid-2024, and pivotal trial planning update expected in Q1

https://www.globenewswire.com/news-release/2024/01/23/2814044/0/en/4DMT-Receives-Rare-Pediatric-Disease-Designation-from-FDA-for-Aerosolized-4D-710-for-Treatment-of-Cystic-Fibrosis-Lung-Disease.html