Our mission is to develop lifelong cures for serious, early-onset rare diseases by combining the best of gene therapy
and gene editing in a one-time treatment.
With GeneRide™, the therapeutic gene is site-specifically integrated into the genome allowing durable gene
expression in dividing cells and mature tissues. Furthermore, therapeutic gene expression is regulated by the
targeted locus so production can be limited to specific cell types.
GeneRide™ is a promoterless and nuclease-free approach to gene insertion which may improve the safety
profile of AAV-based gene therapies.