Selecta Biosciences’ (NASDAQ:SELB) MMA-101 received FDA rare pediatric disease designation for the treatment of methylmalonic acidemia (MMA).
MMA is caused by a mutation in the methylmalonyl-CoA mutase gene, which results in the inability to break down certain proteins and fats. MMA may lead to high blood ammonia levels and is associated with long-term complications including feeding problems, intellectual disability, chronic kidney disease and pancreatitis.
MMA-101 was developed using Selecta’s ImmTOR platform and adeno-associated virus gene therapy vectors developed by its strategic partner, Asklepios BioPharmaceutical.
“When used with AAV gene therapy vectors, Selecta’s ImmTOR aims to inhibit the immune response to the AAV vector, potentially allowing re-dosing of gene therapies,” Carsten Brunn, Ph.D., Selecta’s president and CEO, said in a statement.
“Ongoing clinical programs will focus on evaluating product candidate performance in patients who may have been underdosed or those who may lose transgene expression over time,” he added.
Selecta and Asklepios BioPharma expect to initiate a Phase 1 clinical trial of MMA-101 in the first half of 2021.
