4DMT to Present Interim Data from Aerosolized 4D-710 Phase 1/2 AEROW Clinical Trial for Cystic Fibrosis at 47ᵗʰ European Cystic Fibrosis Conference

Core Viewpoint - 4D Molecular Therapeutics is set to present interim clinical data from the Phase 1/2 AEROW clinical trial for aerosolized 4D-710, aimed at treating cystic fibrosis lung disease, at the 47th European Cystic Fibrosis Conference on June 6, 2024 [1][2] Group 1: Company Overview - 4D Molecular Therapeutics is a clinical-stage genetic medicines company focused on developing therapies for large market diseases, particularly in ophthalmology and pulmonology [6] - The company utilizes a proprietary platform called Therapeutic Vector Evolution to create customized AAV vectors for its product candidates [6] - Currently, 4DMT is advancing five clinical-stage and two preclinical product candidates targeting rare and large market diseases [6] Group 2: Product Information - 4D-710 is designed for aerosol delivery to achieve targeted CFTR expression in lung airway epithelial cells and aims to treat approximately 15% of cystic fibrosis patients whose conditions are not amenable to existing CFTR modulator therapies [4] - The product has received Rare Pediatric Disease Designation and Orphan Drug Designation from the U.S. FDA [5] Group 3: Cystic Fibrosis Context - Cystic fibrosis is a progressive inherited disease affecting nearly 40,000 people in the U.S. and over 105,000 globally, with lung disease being the primary cause of morbidity and mortality [3] - The disease leads to impaired lung function and requires lifelong treatment, often resulting in repeated hospitalizations and increased need for antibiotics [3]