Telomir Pharmaceuticals (TELO) announced new preclinical data demonstrating that its lead candidate, Telomir-1, administered orally, significantly increases telomere length, reverses body weight and muscle loss, and resets cellular aging markers in a validated animal model of Werner Syndrome-a rare genetic disorder also known as adult-onset progeria. These findings confirm and build upon the Company’s previously reported results from a preclinical C. elegans study, which demonstrated that Telomir-1 restored lifespan and normalized physiological decline in animals with a wrn gene mutation. Key Preclinical Findings: DNA methylation is one of the body’s key mechanisms for controlling which genes are turned on or off. It works by attaching small chemical tags to DNA at locations known as CpG islands-which act like gene “on/off” switches. When methylation patterns are intact, cells know which genes to express and when. However, with aging and disease, this regulatory system starts to break down – a phenomenon known as epigenetic drift. In these cases, genes that should be off may turn on inappropriately, and protective genes may be silenced. This malfunction in gene regulation is strongly linked to a wide range of chronic diseases including: Cancer; Neurodegenerative diseases like Alzheimer’s and Parkinson’s; Autoimmune diseases such as lupus and multiple sclerosis; Metabolic conditions like Type 2 diabetes; Premature aging disorders like progeria and Werner Syndrome. In this study, Telomir-1 reversed age-related hypomethylation at two chromosomal regions, restoring methylation patterns to above-normal wild-type levels. This suggests that Telomir-1 may help restore healthy gene regulation and reset the body’s epigenetic aging clock, reducing the risk of dysfunction in key biological systems. Telomeres are the protective caps at the ends of chromosomes that shorten with each cell division and under stress. Telomere shortening is considered one of the hallmarks of aging. In the study, compared with the shortened length in the mutated animals, Telomir-1 increased telomere length by about three-fold. At the higher dose, telomere length significantly exceeded wild-type levels, suggesting not only restoration but also potential enhancement of chromosomal integrity In the wrn-mutant zebrafish model, animals exhibited a 50-60% reduction in body weight and muscle volume. After 14 days of Telomir-1 treatment, these physical markers were restored to levels statistically indistinguishable from healthy controls – indicating functional recovery and metabolic improvement. Reactive oxygen species, which damage cells and accelerate aging, were elevated in untreated animals. Telomir-1 reduced ROS levels by up to 50%, suggesting improved cellular resilience. Roughly 15% of untreated animals died during the 14-day study period, whereas no deaths occurred in any Telomir-1 treated groups – highlighting a systemic survival advantage.
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