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CCB-003 is a disease-modifying gene therapy under development for the treatment of Ehlers-Danlos syndrome, specifically targeting the delivery of a functional type III collagen gene (COL3A1). The therapy aims to address underlying genetic defects by transferring the COL3A1 gene, which encodes collagen type III alpha 1 chain, to restore normal collagen function. Originally developed by Mayo Clinic and now being advanced by Castle Creek Biosciences, CCB-003 is currently in preclinical development. Its primary indication is for congenital disorders affecting skin and musculoskeletal systems, with a focus on Ehlers-Danlos syndrome[1][2].
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