Lead program in Stargardt disease
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.
Additional pipeline across several therapeutic areas
In addition to working in Stargardt disease and other ophthalmological indications, we are developing gene therapy programs in several other therapeutic areas where the platform has also been validated.