Script Biosciences is a biotechnology company developing curative genetic therapies using its proprietary CasPlus gene editing platform and engineered lipid nanoparticle delivery systems to treat debilitating genetic disorders.
Script Biosciences is an early-stage biotechnology company focused on developing curative genetic therapies for diseases with high unmet medical needs. The company utilizes a dual-platform approach combining its proprietary CasPlus gene editing technology with specialized lipid nanoparticle (LNP) delivery vehicles. CasPlus enhances traditional CRISPR systems by integrating an engineered DNA repair enzyme, which allows for precise, predictable gene editing with minimal collateral damage, while the proprietary LNP library enables targeted cargo delivery to vital organs such as the brain, pancreas, and lungs.
These technologies are designed to address the safety and delivery challenges that currently limit the success of genomic interventions. By targeting genetic modifiers associated with triplet repeat disorders, such as Huntington’s disease and myotonic dystrophy type 1, Script Biosciences aims to halt disease progression where few or no therapeutic options exist. The company, which holds a worldwide license for its core technology developed at New York University, is actively engaged in research supported by organizations like the National Science Foundation to advance its gene editing platform toward clinical viability.