GenEdit's delivery vehicle
GenEdit’s proprietary technology can enable the delivery of specific gene editors to a variety of tissues using nanoparticles which are non-viral and polymer-based delivery vehicles.
GenEdit's platform enables broad product opportunity
Genetic diseases are found in various organs including brain, muscle, liver, or blood. GenEdit’s technology platform is based on screening its polymer-based nanoparticle library for the best delivery to the target organs. GenEdit’s proprietary nanoparticles can encapsulate gene therapy molecules and CRISPR proteins.
Our recent publications have demonstrated initial proof-of-concept by exhibiting efficient preclinical gene editing in target tissues, specifically the muscle for Duchenne muscular dystrophy (Nature Biomedical Engineering, 2017) and the brain for Fragile X syndrome (Nature Biomedical Engineering, 2018). In addition, the polymer library has also enabled the delivery of Cpf1 (Cas12a), a next generation CRISPR, in muscle (Nature Communications, 2018).