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Splice Bio

Biotech & Life Sciences · Barcelona, Spain · Founded 2012

Splice Bio is a biotechnology company that specializes in the development of gene therapies for genetic diseases. The company's approach is centered around its proprietary Protein Splicing platform, which is designed to overcome the size limitations of adeno-associated virus (AAV) vectors. This platform enables the delivery of large genes that would otherwise not fit into a single AAV, thereby expanding the range of diseases that can be targeted with gene therapy. The company's business model is focused on the research and development of these novel gene therapies, with the ultimate goal of bringing them to market. Their primary clients are patients suffering from genetic diseases that are currently untreatable or have limited treatment options. Splice Bio operates in the highly specialized and regulated market of genetic medicine. The company's lead program, SB-007, is an investigational gene therapy for the treatment of Stargardt disease, a rare genetic eye disorder that causes progressive vision loss. This indicates a focus on ophthalmology and rare diseases. The company likely generates revenue through partnerships, licensing agreements, and eventually, the sale of its approved therapies. Keywords: gene therapy, protein splicing, AAV vectors, genetic diseases, Stargardt disease, ophthalmology, rare diseases, biotechnology, genetic medicine, large gene delivery