Gene Therapy Receives Rare Pediatric Disease Designation for Methylmalonic Acidemia


There are currently no approved therapies for methylmalonic acidemia, a rare and life-threatening disease caused by mutations in the MUT gene.

Officials with the FDA have granted rare pediatric disease designation to LogicBio Therapeutics’ LB-001, an investigational gene-editing treatment for methylmalonic acidemia (MMA), according to a press release.

The agency grants rare pediatric disease designation for serious and life-threatening diseases that primarily affect children through age 18 and affect fewer than 200,000 individuals in the United States. LogicBio also previously received orphan drug designation from the FDA for LB-001.

LB-001 is a recombinant adeno-associated viral vector with human methylmalonyl-COA mutase (MUT) gene designed for the treatment of pediatric patients with MMA, a rare and life-threatening disease for which there are no approved therapies.

MMA, which usually starts in early childhood, is primarily caused by mutations in the MUT gene. With MMA, the body is unable to properly break down certain fats and proteins. This can result in a toxic buildup of metabolites that can lead to significant morbidity and mortality, including infections, neurodevelopmental disabilities, and chronic kidney diseases.

The gene therapy is based on LogicBio’s GeneRide technology, which enables site-specific integration and lifelong expression of therapeutic transgenes without the use of exogenous promoters or nucleases. According to LogicBio, the therapy is designed to incorporate a functioning version of the faulty MUT gene into the patients’ genome.

In preclinical studies conducted at the National Institutes of Health, hepatic correction was durable over 1 year and suggestive of a selective growth advantage.

“MMA is a life-threatening rare disease that presents as early as the first week of life and has no approved therapies,” Fred Chereau, chief executive officer of LogicBio, said in a statement. “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.”

According to Chereau, the company expects to file an Investigational New Drug application with the FDA in the fourth quarter of 2019.


LogicBio Therapeutics Receives Rare Pediatric Disease Designation for LB-001 for the Treatment of Methylmalonic Acidemia [news release]. LogicBio. Accessed July 18, 2019.

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