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SIG-205

Development stage: Preclinical
Lead developer: Eli Lilly
Modality: Cell Therapies, Recombinant Proteins and Enzymes, Gene Therapies

Overview

**SIG-205** is an investigational cell therapy consisting of cultured human retinal pigment epithelial cells (ARPE-19) genetically modified using a non-viral vector to express human native alpha-L-iduronidase enzyme (hIDUA). The cells are encapsulated in two-layer modified alginate spheres to protect them from immune rejection while enabling sustained enzyme delivery. Developed by Sigilon Therapeutics, it targets **mucopolysaccharidosis type I (MPS-1)**, a lysosomal storage disorder caused by IDUA deficiency leading to glycosaminoglycan accumulation, skeletal deformities, and other complications. The therapy aims to provide long-term enzyme replacement via localized secretion in a shielded cellular platform.[1][4][5]

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