SIG-005

SIG-005 is an encapsulated, non-viral cell-based gene therapy developed for the treatment of mucopolysaccharidosis type I (MPS-I), a rare lysosomal storage disorder. The therapy consists of a human cell line genetically modified with a non-viral vector to express human alpha-L-iduronidase (IDUA), the enzyme deficient in MPS-I patients. Encapsulated within biocompatible spheres using Sigilon’s Shielded Living Therapeutics platform, these engineered cells are designed to provide sustained and controllable delivery of IDUA, aiming to reduce glycosaminoglycan accumulation and address multi-organ involvement characteristic of MPS-I. The approach is intended as a durable, redosable alternative to current therapies such as enzyme replacement or stem cell transplantation[1][2][5][8].