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Muscular Dystrophy Technology
3 companies found
Technologybased inGaithersburg, MASSACHUSETTS (USA)
In November 2023, Selecta Biosciences Inc. (NASDAQ: SELB) merged with Cartesian Therapeutics to create a publicly traded company pioneering RNA cell therapy to treat autoimmune disease. The combined company will focus on advancing Cartesian’s wholly ...
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 ...
Technologybased inBoston, MASSACHUSETTS (USA)
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — ...
Technologybased inNess Ziona, ISRAEL
Science in Action (SIA) is a boutique preclinical contract research organization (CRO) founded in 2010 by immunologist Raanan Margalit. SIA is GLP-accredited for toxicology studies in multiple areas. SIA has extensive experience in tailor-made ...
SIA's philanthropic research supports the Israeli Duchenne Parent Association. This is an eort to develop a cure for Duchenne Muscular Dystrophy (DMD). DMD is the most common, fatal genetic disorder diagnosed in ...