Safety and initial activity of autologous human B cells genetically engineered to express human iduronidase using the Sleeping Beauty transposon system. Results from a first-in-human clinical trial in subjects with MPS I SAN FRANCISCO, Jan. 27, 2025 /PRNewswire/ — Immusoft of CA, a cell therapy…

First-in-human clinical trial of genetically engineered B cells: Application to the treatment of mucopolysaccharidosis type I

Pharmacodynamic effects and functional improvements were observed through nine months post-dosing with a single administration of ISP-001. As of the data cutoff date of September 19, 2024, no adverse events have been reported. Data for ISP-001 for Mucopolysaccharidosis Type I to be presented on October…