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Model MMA-101 - ImmTORTM + Gene Therapy Candidate for Methylmalonic Acidemia (MMA)
Selecta’s first ImmTORTM + gene therapy candidate MMA-101 for methylmalonic acidemia (MMA) is expected to enter clinical trials the end of 2021. MMA is a rare metabolic disease that may lead to metabolic acidosis and hyperammonemia and is associated with long-term complications including feeding problems, developmental delay, intellectual disability, and chronic kidney disease.
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Additional programs, in preclinical development, are Pompe disease (in partnership with AskBio) and Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (both in partnership with Sarepta).