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SR-GT1

Development stage: Preclinical
Lead developer: StemRIM
Modality: Gene Therapies, Stem Cell Therapies → Cell Therapies
Administration: Intralesional

Overview

SR-GT1 is an ex vivo gene therapy being developed by StemRIM for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB). The therapy involves the extraction of patient-derived mesenchymal stem cells (MSCs), which are then transduced with a lentiviral vector to express the COL7A1 gene, encoding Type VII collagen. These genetically modified cells are subsequently administered via intrablister injection. The goal is to restore the production of Type VII collagen, which is deficient in RDEB patients, thereby improving skin integrity and reducing blister formation. It is currently in the preclinical stage of development.

Targets

COL7A1 (Collagen type VII alpha 1 chain)

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