Bringing gene therapy to pediatric diseases
Gene therapy has advanced steadily over four decades and is now a therapeutic reality in select adult indications. For many childhood diseases, however, gene therapy remains tantalizingly out of reach. Current gene therapy approaches provide a functioning copy of the missing or faulty gene to the cell. In the rapidly dividing cells of children these episomal genes are diluted, resulting in a loss of therapeutic effect.
LogicBio has assembled a unique set of technologies united around one goal: to bring the benefits of gene therapy to the fight against childhood diseases. Our initial focus is on rare liver disorders where GeneRide™ enables a durable treatment effect as the liver grows and divides. LogicBio is also exploring other tissues where our technology could be applied.
LogicBio's broad pipeline is built on two core technologies designed to address the major shortcomings of traditional gene editing and gene therapy approaches. We combine:
- GeneRide™, a promoterless, nuclease-free genome editing technology, drives high expression of therapeutic proteins by harnessing homologous recombination—a naturally occurring process—to safely integrate transgenes site-specifically into the genome to provide a durable cure from a one-time treatment; and
- Our proprietary library of synthetic, non-pathogenic, recombinant adeno-associated viral (rAAV) vectors developed at Stanford University designed to improve the predictability of vector performance in clinical trials.