LogicBio Therapeutics, Inc. (Nasdaq: LOGC), a genome editing company focusing on developing medicines for the long-term treatment of rare diseases in pediatric patients, today announced that it has submitted an application with the U.S. for an Investigational New Drug (IND). Food and Drug Administration (FDA) to launch a phase 1/2 trial of LB-001, the human methylmalonyl-COA mutase (MMUT) recombinant adeno-associated viral vector for the treatment of methylmalonic acidemia (MMA). LB-001 leverages the patented, promoterless, nuclease-free genome editing technology GeneRideTM from LogicBio and has previously received FDA designations for both orphan drugs and rare pediatric diseases.
Once the FDA approves the IND, LogicBio plans to release additional details about the proposed Phase 1/2 study, including sample duration, endpoints, and timelines. In the first half of 2020, LogicBio plans to launch a Phase 1/2 trial during pediatric MMA patients, with preliminary data planned for the second half of 2020.
LogicBio today also addressed key recent and forthcoming milestones.
In a second indication, Crigler-Najjar Syndrome, we formed a partnership with Takeda to exploit the GeneRide platform. LogicBio and Takeda will further study and improve LB-301, an investigational pediatric genome editing therapy based on the GeneRide technology used by LogicBio to treat CN. Review of Natural History Retrospective conducted at MMA. The purpose of this study is to evaluate the progression of disease in pediatric patients (born since 2010) with severe MMA, in order to inform LogicBio’s future development in MMA and its discussions with regulatory agencies.
Positive results published at the European Society of Gene and Cell Therapy Annual Meeting on Next Generation Capsid Development Program. Evidence from a group of novel, adeno-associated synthetic virus (AAV) capsid candidates tested against references AAV2, AAV8, and LK-03 were presented. Both capsids displayed selective tropism and more efficient transduction and gene expression than a chimeric FRG mouse model’s reference capsides in human hepatocytes.
Doubling of the available lab and office space to support the development and expansion of GeneRide software capabilities. In the spring of 2020, LogicBio plans to move to new headquarters in Lexington, Mass. LogicBio will add vivarium space, internal capacity for production, and increase its capacity to produce preclinical material in-house.
About Methylmalonic Acidemia
Methylmalonic acidemia is a rare, life-threatening, autosomal recessive disease that is primarily caused by mutations in the MMUT gene and for which no approved therapies exist. The disorder, which starts in the first month of life, prevents the body from adequately processing some fats and proteins, resulting in a toxic accumulation of metabolites that in infants and children may cause life-threatening decompensations.
LB-001 is a pediatric genome-editing investigational therapy based on GeneRideTM technology from LogicBio. GeneRide allows for site-specific integration and lifelong expression of therapeutic transgenes, without using exogenous promoters or nucleases. LB-001 has been granted U.S. designations for both orphan drugs and rare pediatric diseases. Meat and Medication Administration.
About LogicBio Therapeutics
LogicBio Therapeutics is a genome editing company that uses GeneRideTM, its proprietary technology platform, to create medicines to durably treat rare diseases in pediatric patients with major unmet medical needs. GeneRide enables the site-specific incorporation of a therapeutic transgene into a nuclease-free and promotion-free system by relying on the homologous native recombination mechanism to drive future lifelong expression.