The Science

LogicBio is a breakthrough gene therapy company founded on two core platform technologies. First is GeneRide™, a promoterless, nuclease-free genome editing technology that addresses key shortcomings in current gene therapy approaches. GeneRide™ achieves high expression of therapeutic proteins by harnessing homologous recombination to integrate transgenes site-specifically into the genome to provide a durable cure from a one-time therapy.The company also has access to a library of synthetic, non-pathogenic, recombinant adeno-associated viral (rAAV) vectors developed at Stanford University that provide better predictability of vector performance in clinical trials.

The Patients

There are thousands of rare genetic disorders that affect children. Gene therapy could potentially cure many of these diseases by permanently expressing a functional version of the missing or faulty gene. However, a major challenge is the proliferation of cells in the growing tissue of a child which dilutes the benefit of the therapy. In many pediatric diseases symptoms arise in the first year of life and progress rapidly, often leading to death. Intervening early is critical to provide transformational medicines for patients and families with limited options. LogicBio, with its proprietary technology, is uniquely positioned to deliver a durable cure for life-threatening genetic pediatric diseases.