Splice Therapeutics

SpliceBio is a biotechnology company based in Barcelona that specializes in developing next-generation gene therapies to treat genetic diseases. The company's proprietary Protein Splicing platform utilizes engineered inteins to overcome the cargo capacity limitations of Adeno-Associated Virus (AAV) vectors, enabling the delivery of large genes. Their lead program, SB007, targets Stargardt disease, a common form of inherited macular degeneration caused by mutations in the large ABCA4 gene. By reconstituting full-length proteins from two separate AAV vectors, SpliceBio aims to address previously undruggable genetic conditions. The company is backed by a strong syndicate of international life science investors and is expanding its pipeline into other therapeutic areas.