4D Molecular Therapeutics is a clinical‐stage biotechnology company focused on the discovery and development of targeted, engineered adeno‐associated virus (AAV) gene therapies. Leveraging its proprietary Therapeutic Vector Evolution platform, the company designs novel AAV capsids optimized for tissue specificity and durability of gene expression. These capsids are paired with tailored genetic payloads to address a range of inherited and degenerative diseases, including ophthalmic disorders and neuromuscular conditions.
The company’s pipeline includes candidates for X-linked retinitis pigmentosa, achromatopsia, Leber hereditary optic neuropathy and Duchenne muscular dystrophy, among others. Preclinical and early clinical programs aim to demonstrate improved safety and efficacy profiles by maximizing delivery to target cells while minimizing off‐target exposure. 4D’s technology is intended to overcome key limitations of first‐generation gene therapies, such as immune responses to viral capsids and insufficient gene expression in certain tissues.
Founded in 2013 and headquartered in the San Francisco Bay Area, 4D Molecular Therapeutics operates research and development facilities in California and collaborates with manufacturing partners across North America and Europe. The company’s integrated approach combines in silico, in vitro and in vivo screening to rapidly advance lead candidates toward clinical evaluation. Through strategic partnerships with established biopharmaceutical firms, 4D seeks to accelerate development timelines and expand global reach for its therapeutic programs.
The senior management team comprises industry veterans with extensive experience in gene therapy, molecular biology and biopharmaceutical development. Under the leadership of its executive team, 4D has secured multiple alliances and research collaborations to strengthen its platform capabilities and support late‐stage clinical studies. As it advances its portfolio toward pivotal trials, the company continues to attract investment and scientific talent focused on transforming the treatment landscape for serious genetic diseases.
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