LogicBio Therapeutics Receives Rare Pediatric Disease Designation for LB-001 for the Treatment of Methylmalonic Acidemia
“MMA is a life-threatening rare disease that presents as early as the first week of life and has no approved therapies. The urgency of our work with LB-001, our lead program, is underscored by both this new rare pediatric disease designation and the previously granted orphan drug designation. These designations signal the clear unmet need for transformative treatments for MMA patients,” said
LB-001 is an investigational pediatric genome editing therapy based on LogicBio’s GeneRide™ technology. GeneRide enables site-specific integration and lifelong expression of therapeutic transgenes, without the use of exogenous promoters or nucleases. LB-001 is designed to incorporate a functioning version of the faulty MUT gene into the genome of MMA patients. LogicBio has demonstrated preclinical proof-of-concept of GeneRide in multiple animal models of the disease, improving survival and reversing disease pathology. In preclinical MMA models, LogicBio has shown that cells into which GeneRide has inserted a transgene demonstrate a selective survival advantage over cells not expressing the transgene.
Primarily caused by mutations in the MUT gene, MMA is a rare, life-threatening, autosomal recessive disease that starts in early childhood for which there are no approved therapies. The disease prevents the body from properly processing certain fats and proteins, resulting in a toxic accumulation of metabolites that can cause life threatening decompensations in infants and children. This buildup can lead to significant morbidity and mortality, including infections, neurodevelopmental disabilities and chronic kidney disease.
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Associate Director, Investor Relations
Ten Bridge Communications
Source: LogicBio Therapeutics