Few treatment options exist for patients suffering from genetic disorders, which often manifest earlier in life, cause severe symptoms, and shortened lifespan. Genetic therapies offer the potential to precisely correct diverse genetic
disorders. However, safety of genomic interventions and delivery to target organs remain key challenges.
Script Biosciences was founded with a singular focus of developing curative therapies by safely and effectively correcting disease pathologies arising from mutations in the genome. Our therapeutic approach is grounded in the synergy of two cutting-edge platform technologies: a CRISPR-based gene editing system (CasPlus) and a specialized family of engineered lipid nanoparticle carriers. These vehicles facilitate targeted cargo delivery to vital organs such as the brain, pancreas, or lungs, forming the cornerstone of our treatment strategy.
LNPs are clinically proven mRNA delivery vehicles in the liver and immune cells. However, delivery to other organs has been a challenge. Our proprietary LNP system consists of a unique library of helper and ionizable lipids, which in conjunction with specific delivery modes, enable targeted delivery to organs such as the brain and pancreas.
The CasPlus platform pairs Cas nucleases with an engineered DNA repair enzyme to achieve high editing efficiency while minimizing unwanted genomic modifications. CasPlus can potentially work with different Cas nucleases to enable the correction of diverse mutations. CasPlus has wide applicability and superior editing efficiency compared to existing technologies with the ability to produce 1 -3 base pair insertions in a predictable and template-free approach. These attributes uniquely position CasPlus within the toolbox of gene editing technologies.
Our proprietary pipeline focuses on targeting disease modifying mechanisms associated with triplet disease disorders.
These disorders are the result of anomalous expansion of repetitive sequences resulting in pathologies primarily in the central nervous system and in some cases the muscle. We are currently targeting the mismatch repair gene MSH3 which is a genetic modifier of the CAG/CTG repeat expansion associated with diseases such as Huntington’s disease (HD) and myotonic dystrophy type 1 (DM1). Our therapeutic goal is to halt disease progression by inactivating MSH3 and stop further expansion of the repeats.
