CasPlus

CasPlus is a novel CRISPR/Cas-based gene editing platform technology developed by **Script Biosciences**. It integrates an engineered **T4 DNA polymerase** with **Cas9 nuclease** and guide RNA to enable high-efficiency, exogenous template-free DNA repair of double-strand breaks. The technology is designed as a biologic/gene therapy modality that prevents large on-target DNA deletions and chromosomal rearrangements, offering a safer alternative to traditional CRISPR/Cas9 systems. CasPlus is being developed to treat genetic diseases including **Duchenne muscular dystrophy (DMD)** and **cystic fibrosis (CF)**. For DMD, it works by restoring the reading frame of dystrophin genes with frameshift mutations in exons 51 and 53. For CF, it can correct the F508del mutation in the CFTR gene that causes over 80% of cystic fibrosis cases. Script Biosciences is currently advancing the lead DMD program toward IND-enabling studies.