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Single intravenous administration of mLB-001 in neonatal or adult MMA mice correlated with prevention of severe weight loss and mortality when challenged with a high protein diet.
LEXINGTON, Mass., Sept. 21, 2022 /PRNewswire/ -- LogicBio Therapeutics, Inc. (NASDAQ:LOGC), a clinical-stage genetic medicine company, today announced that results from the company's preclinical research study entitled "Novel AAV-mediated genome editing therapy prevents metabolic decompensation in a mouse model of methylmalonic acidemia" have been published in the peer-reviewed journal PLOS ONE.
MMA is a metabolic disorder most commonly caused by mutations in the methylmalonyl-CoA mutase (MMUT) gene. Patients with MMUT deficiency are currently treated with strict life-long dietary management to mitigate acute illness that worsens the patient's basal condition, particularly with respect to metabolic brain injury. Early-stage research has indicated that restoration of Mmut enzymatic activity by gene addition mediated by recombinant adeno-associated virus (rAAV) could be a promising approach in the treatment of MMUT deficiency in pediatric patients.
In the study, researchers developed a novel protein-controlled diet regimen in a MMUT deficient mouse model of MMA that mimics the metabolic crises that MMA patients experience. Mice were treated with a single administration of mLB-001, a nuclease-free, promoterless recombinant AAV vector designed based on LogicBio's proprietary GeneRide® platform to deliver the mouse MMUT into the endogenous albumin locus. Mice deficient in MMUT that were treated with the Generide AAV vector had attenuated body weight loss and were protected from mortality when challenged with a high protein diet.
GeneRide-edited hepatocytes also expressed functional MMUT protein and expanded over time in the MMUT deficient mice, suggesting a selective growth advantage over the diseased cells. The expansion of the edited cells was detected over time in the MMA mice by measuring increasing levels of Alb-2A, the same technology-specific biomarker that is being used in LogicBio's SUNRISE trial, a first-in-human, open-label, multi-center, Phase 1/2 clinical trial designed to assess the safety, tolerability, and preliminary efficacy of a single intravenous infusion of LB-001 in pediatric patients with MMA.
"There are no approved therapies to treat the underlying cause of MMUT deficiency. Currently, pediatric patients are managed with strict life-long dietary restrictions designed to help mitigate acute illness that worsens the patient's basal condition, particularly with respect to metabolic brain injury," said Mariana Nacht, chief scientific officer at LogicBio. "These new preclinical results provide strong additional support for the continued development of LB-001 in this indication with the potential to deliver significant benefits to pediatric patients."
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Posted In: LOGC
