Also sAAVy platform
LogicBio Therapeutics is developing genetic medicines to fight against rare pediatric diseases. LogicBio is pioneering safer, site-specific genome editing technology to provide durable therapies using their GeneRide proprietary platform. LogicBio has currently designed GeneRide to identify liver-targeted therapies - the platform uses a synthetic viral vector to deliver the corrective transgene to a patient's cell into the highly-expressed albumin locus. By using a 2A peptide, both the corrective transgene and albumin are produced as separate protein products and modify albumin with a small tag to monitor GeneRide activity. LogicBio’s initial product candidates will address diseases by targeting the liver, but the company is looking to expand to other tissues.