SpliceBio products

SpliceBio specializes in addressing the challenges of gene therapy by leveraging engineered split inteins. Traditional adeno-associated virus (AAV) vectors are limited by size, restricting their ability to package only small genes, thus leaving many monogenic diseases without effective treatment solutions. The company has focused on the innovative application of inteins, a class of proteins that can perform protein splicing through biochemical reactions involving cleavage and bond formation. Historically, natural inteins presented several limitations, making them unsuitable for human therapeutics. SpliceBio's co-founders have overcome these barriers by developing advanced engineered split inteins at Princeton, tailored specifically for therapeutic applications. These advancements form the basis of their proprietary Protein Splicing platform, enabling the development of novel gene therapies targeting a variety of therapeutic areas, offering potential breakthroughs for patients with unmet medical needs.

SpliceBio is developing several gene therapy programs aimed at treating genetic diseases by exploiting our proprietary Protein Splicing platform. Our lead program is in Stargardt disease, the most common inherited retinal disorder caused by mutations in a single gene, ABCA4, which at 6.8 kb is too large for single AAV vectors.