This segment focuses on developing gene therapies for inherited retinal diseases and age-related macular degeneration (AMD). 4DMT leverages its Therapeutic Vector Evolution platform to create AAV vectors that efficiently target retinal cells, delivering therapeutic genes to correct genetic defects or inhibit angiogenesis. Key programs include 4D-125 for X-linked retinitis pigmentosa (XLRP) and 4D-150 for wet AMD, both in clinical development. The goal is to provide long-lasting vision improvement or stabilization for patients with limited treatment options. The company is advancing 4D-150 into Phase 3 clinical trials, demonstrating its commitment to addressing significant unmet needs in ophthalmology. Partnerships and collaborations are essential for expanding the reach and impact of these therapies.

The cardiology segment is dedicated to developing gene therapies for heart diseases with a focus on Fabry disease. 4DMT's approach involves using targeted AAV vectors to deliver functional genes to cardiac cells, aiming to correct the underlying genetic cause of the disease and improve heart function. The lead program, 4D-310, is in Phase 1/2 clinical trials for Fabry disease. This segment emphasizes the development of precision medicines that can provide durable therapeutic benefits for patients with severe cardiac conditions. Future opportunities include expanding the pipeline to address other genetic cardiomyopathies and exploring novel delivery methods to enhance gene transfer efficiency and safety. Collaborations with research institutions and pharmaceutical companies are crucial for advancing these innovative therapies.

This segment is focused on developing gene therapies for cystic fibrosis (CF) lung disease. 4DMT is utilizing its Therapeutic Vector Evolution platform to engineer AAV vectors that can effectively deliver functional CFTR genes to lung cells, addressing the underlying cause of CF. The lead candidate, 4D-710, is an IND candidate for the treatment of cystic fibrosis lung disease. The goal is to improve lung function and reduce the frequency of pulmonary exacerbations in CF patients. This segment involves extensive preclinical research and development to optimize vector design, delivery methods, and immune responses. Partnerships with the Cystic Fibrosis Foundation (CFF) and other organizations are essential for accelerating the development of these potentially life-changing therapies.