Gene and Cell Therapies in Focus at ASGCT 2025: Tackling Rare Diseases, Autoimmune Conditions, and More

Immusoft, a California-based cell therapy innovator, is presenting the world’s first clinical trial data for engineered B cells targeting mucopolysaccharidosis type I (MPS I), a rare genetic disorder caused by a deficiency in the enzyme iduronidase. This condition triggers a harmful buildup of substances, leading to severe developmental and physical challenges. At ASGCT 2025, Paul J. Orchard, MD, from the University of Minnesota, will share results from a first-in-human trial, highlighting the safety and early efficacy of autologous B cells genetically modified to produce iduronidase. This novel approach could transform therapeutic protein delivery and offers a promising path for MPS I patients. Read More>>