Opus Genetics (NASDAQ:IRD) is a biotechnology company focused on the development of gene therapies for rare and inherited diseases. Leveraging proprietary vector design and delivery technologies, the company aims to address unmet medical needs in areas such as inherited retinal disorders, metabolic deficiencies and neuromuscular conditions. Opus Genetics’ platform integrates advanced viral vector engineering, next-generation sequencing and functional genomics to optimize tissue-specific gene expression and therapeutic efficacy. The company’s core mission is to translate cutting-edge genetic research into safe and durable treatments for patients worldwide.
Founded in 2015 and headquartered in Cambridge, Massachusetts, Opus Genetics has established research collaborations with leading academic institutions in North America and Europe. The firm operates state-of-the-art laboratories where its scientists develop and validate adeno-associated virus (AAV) and lentiviral vector systems. In addition to its internal pipeline, Opus Genetics offers custom vector production and process development services to biopharmaceutical partners, supporting preclinical studies and early-stage clinical programs. This dual approach—advancing proprietary assets while serving external clients—positions the company as both innovator and CDMO (contract development and manufacturing organization) in the gene therapy landscape.
Over the years, Opus Genetics has built a diversified pipeline, with lead candidates targeting retinal dystrophies and metabolic enzyme deficiencies. Preclinical data have demonstrated durable gene expression and favorable safety profiles in animal models, paving the way for planned IND (Investigational New Drug) filings. The company’s R&D team includes experts in molecular biology, immunology and regulatory affairs, many of whom bring experience from established biotechs and academic gene therapy centers. Strategic partnerships with patient advocacy groups further guide Opus Genetics’ clinical development strategy and patient engagement efforts.
Under the leadership of Chief Executive Officer Dr. Laura Mitchell and Chief Scientific Officer Dr. Alejandro Torres, Opus Genetics has expanded its global footprint with research offices in London and Tokyo. The executive team combines deep scientific know-how with commercial expertise, ensuring that the company’s gene therapies meet rigorous regulatory standards and address real-world patient needs. As Opus Genetics advances its key programs toward human trials, it remains committed to applying its technology platform to a broader range of genetic diseases, with the goal of delivering transformative treatments to patients and establishing itself as a leader in the gene therapy industry.
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