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AR-001 is a preclinical gene therapy candidate developed by Bavarian Systems and Technologies GmbH (BST BIO). It utilizes short hairpin RNA (shRNA) to target and silence the expression of the androgen receptor (AR). By reducing AR levels, the therapy aims to treat conditions driven by androgen signaling, specifically androgenetic alopecia (pattern hair loss) and androgen-dependent prostate cancer. The shRNA approach provides a mechanism for long-term suppression of the target protein compared to traditional small molecule inhibitors, potentially offering a more durable therapeutic effect by addressing the root cause of androgen-mediated pathology at the genetic level.
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