Telomir announced new preclinical data showing that its lead candidate, Telomir-1, prevented cellular aging in human progeria cell lines obtained from the Progeria Research Foundation. Progeria, or Hutchinson-Gilford Progeria Syndrome, is an ultra-rare pediatric disorder caused by a mutation in the LMNA gene. This mutation results in the production of an abnormal protein called progerin, which drives rapid biological aging in children. Telomir-1 is designed to regulate intracellular metal ions, reduce oxidative stress, restore mitochondrial function, extend telomere length, reverse muscle loss, and reset age-associated DNA methylation patterns – all of which are critical biological pathways implicated in progeria and broader age-related diseases. In this study, conducted by Smart Assays, Telomir-1 was tested in cells taken directly from a child with HGPS. These cells were obtained from The Progeria Research Foundation. The study evaluated cell viability, reactive oxygen species, and intracellular calcium signaling – a marker of mitochondrial dysfunction – under both normal and stress-induced conditions. Key findings include: Telomir-1 increased survival in a dose-dependent manner, both under basal conditions and even under stress conditions induced by copper and iron-two metal ions known to accelerate aging by generating oxidative damage and destabilizing DNA and telomeres. Progeria cells exhibited abnormally high levels of reactive oxygen species, a hallmark of cellular aging. Telomir-1 normalized these levels, both under basal conditions and even when ROS was further elevated by toxic metal exposure. Iron-induced calcium overload – a signal of mitochondrial damage and a known feature of HGPS – was significantly reduced with Telomir-1, indicating restored mitochondrial regulation and improved cellular energy balance. These results demonstrate that Telomir-1 directly addresses the core cellular dysfunctions driving disease features in progeria – not only protecting cells from damage but restoring critical biological functions. The fact that these results were observed in actual patient-derived human cells offers strong early validation of Telomir-1’s therapeutic potential.
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