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Gene Therapy Program Equipment & Supplies In USA
4 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 ...
Manufactured by:SpliceBio based inBarcelona, SPAIN
SpliceBio is developing several gene therapy programs aimed at treating genetic diseases by exploiting our proprietary Protein Splicing platform. Our lead program is in Stargardt disease, the most common inherited retinal disorder caused by mutations in a single gene, ABCA4, which at 6.8 kb is too large for ...
Manufactured by:SpliceBio based inBarcelona, SPAIN
SpliceBio specializes in addressing the challenges of gene therapy by leveraging engineered split inteins. Traditional adeno-associated virus (AAV) vectors are limited by size, restricting their ability to package only small genes, thus leaving many monogenic diseases without effective treatment solutions. The company has focused on the innovative application of inteins, a class of proteins that ...