Cartesian Therapeutics
2 products found

Cartesian Therapeutics products

ImmTOR + Gene Therapy Program

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.

Gene Therapy Program for the Treatment of Ornithine Transcarbamylase (OTC)

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. Severe symptoms include inability to control body temperature and breathing rate, seizures, coma, developmental delays, and intellectual disability. Less severe forms of the disorder are characterized by delirium, erratic behavior, aversion to high protein foods, vomiting and seizures.