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Gene Therapy Program Equipment Supplied In Usa
2 equipment items found
by:Cartesian Therapeutics based inGaithersburg, MASSACHUSETTS (USA)
Selecta’s wholly owned gene therapy program for the treatment of ornithine transcarbamylase (OTC) deficiency is expected to enter the clinic in 2022. OTC deficiency is a genetic disorder urea cycle that causes ammonia to accumulate in the blood. The most severe form of the disorder presents within the first few days of life. ...
by:Cartesian Therapeutics based inGaithersburg, MASSACHUSETTS (USA)
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 ...