LogicBio Therapeutics Receives FDA Orphan Drug Designation for LB-001 for the Treatment of Methylmalonic Acidemia
“We are pleased to receive orphan drug designation for LB-001, our lead program in development for patients with methylmalonic acidemia. This regulatory distinction underscores the need for revolutionary treatments for MMA patients,” said
Orphan drug designation is granted by the
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.
About LogicBio Therapeutics
LogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical needs using GeneRide™, its proprietary technology platform. GeneRide enables the site-specific integration of a therapeutic transgene in a nuclease-free and promoterless approach by relying on the native process of homologous recombination to drive lifelong expression. Headquartered in Cambridge, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.
For more information, please visit www.logicbio.com.
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Source: LogicBio Therapeutics