Design Therapeutics is a clinical‐stage biopharmaceutical company specializing in the development of gene therapies for rare neuromuscular disorders. The company’s proprietary platform leverages adeno‐associated virus (AAV) vectors to deliver therapeutic genes or gene regulators to target muscle tissue. Design Therapeutics’ lead program, DT-010, is designed to address the underlying cause of Duchenne and Becker muscular dystrophy by restoring functional dystrophin protein expression in skeletal and cardiac muscle cells.
Beyond Duchenne and Becker muscular dystrophy, Design Therapeutics is advancing additional pipeline candidates aimed at other genetic muscle diseases. A second program focuses on facioscapulohumeral muscular dystrophy (FSHD) by employing an AAV‐based strategy to silence the DUX4 gene implicated in disease progression. The company is also exploring treatments for myotonic dystrophy type 1 (DM1), applying the same vector technology to deliver oligonucleotide or gene editing payloads that modulate pathogenic RNA repeats.
Founded in 2020 and headquartered in Boston, Design Therapeutics has rapidly assembled a team of experts in gene therapy, neuromuscular biology and clinical development. Chief Executive Officer Michel Perron, Ph.D., brings more than two decades of experience in rare disease research and biopharmaceutical leadership. The company’s scientific advisory board features key opinion leaders in neuromuscular disorders, supporting the design and execution of clinical trials across multiple patient populations.
Design Therapeutics conducts its research and early‐stage clinical studies in the United States, with plans to expand global trial sites to Europe and Asia as its development programs progress. Through collaborations with academic centers and patient advocacy groups, the company aims to accelerate enrollment, gather comprehensive natural history data and ensure broad access to investigational therapies. Its long‐term strategy encompasses both in‐house manufacturing scale-up and partnerships with contract development and manufacturing organizations to support future commercialization efforts.
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