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SRN-22 is a preclinical-stage antisense oligonucleotide (ASO) developed by Sirana Pharma for the treatment of Osteogenesis Imperfecta (OI), a genetic disorder characterized by fragile bones. The therapeutic candidate utilizes Sirana's proprietary Musculoskeletal Targeting Technology (MTT), which conjugates the ASO to a bone-targeting peptide (typically a sequence of aspartic acid residues) to facilitate selective delivery to bone tissue. This targeted approach is designed to increase the local concentration of the drug in the skeleton while minimizing systemic off-target effects. SRN-22 aims to modulate the expression of specific genetic regulators of bone metabolism to improve bone mineral density and skeletal integrity in patients with OI.
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