Next Generation
Next Generation
GENOMIC MEDICINE
GENOMIC MEDICINE
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.
Our Platforms
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.
Our Pipeline
Our proprietary pipeline focuses on targeting disease modifying mechanisms associated with triplet disease disorders.
*Pipeline represents diseases with high unmet needs, few therapeutic options and a combined market size in excess of $10 Billion
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.
script linkedin
Script Biosciences focuses on positively impacting lives of patients suffering from genetically driven debilitating diseases. We are aiming for potential cures for such diseases by harnessing the power of our proprietary gene editing platform. Follow our Linkedin to stay up to date with what our team is working on.
Presentations & Publications
View our team’s recent published work
