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GF-3001 is a formulation of an adeno-associated viral (AAV) vector-based gene therapy developed by Genflow Biosciences. It is designed to deliver a transgene encoding the cDNA portion of a variant of the human SIRT6 gene, specifically one found in centenarians, to dermal fibroblasts. The therapy aims to modulate Sirtuin 6 (SIRT6) activity, which is associated with DNA maintenance and cellular longevity. The primary indication under development for GF-3001 is Werner Syndrome, a rare genetic disorder characterized by premature aging. As of early 2025, the drug remains in the discovery or preclinical stage with no clinical trial data yet reported[1][2][3][4][5].
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