Latest news with #Telomir-1
Miami Herald
4 days ago
- Health
- Miami Herald
Telomir Pharmaceuticals Prevents Cellular Aging in Patient-Derived Cells from Children with Progeria - an Ultra-Rare Genetic Disorder that Causes Rapid Aging
Study used cell lines obtained from the Progeria Research Foundation to evaluate Telomir-1's effects on key drivers of accelerated aging MIAMI, FLORIDA / ACCESS Newswire / June 18, 2025 / Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), a preclinical-stage biotechnology company focused on reversing biological aging and age-related diseases, today announced compelling 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 (HGPS), 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. There are an estimated 400-500 known cases worldwide, including fewer than 30 children currently living with the disease in the United States. Symptoms typically begin within the first two years of life and include growth failure, joint stiffness, loss of body fat and hair, and severe cardiovascular disease. Children with progeria have an average life expectancy of just 13 to 15 years, with most dying from heart attacks or strokes at a young age. The only FDA-approved therapy for progeria, Zokinvy® (lonafarnib), is a farnesyltransferase inhibitor that has been shown to extend lifespan by an average of 4.3 years. However, Zokinvy does not reverse the underlying disease pathology or halt cardiovascular deterioration, which remains the leading cause of death. No approved therapy restores normal cell function or reverses the biological hallmarks of accelerated aging in progeria, highlighting a significant and urgent unmet medical need. 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 (ROS), and intracellular calcium signaling - a marker of mitochondrial dysfunction - under both normal and stress-induced conditions. Key findings include: Improved cell viability: 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 of oxidative stress: Progeria cells exhibited abnormally high levels of reactive oxygen species (ROS), a hallmark of cellular aging. Telomir-1 normalized these levels, both under basal conditions and even when ROS was further elevated by toxic metal of mitochondrial function: 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. "These results provide the strongest evidence to date that Telomir-1 is not only protective but also restorative at the molecular and cellular level," said Dr. Angel, Chief Scientific Advisor of Telomir. "What's especially promising is that the improvements we observed directly target the mechanisms known to drive disease progression in progeria - oxidative stress, metal toxicity, and mitochondrial instability. This level of functional rescue in actual patient-derived cells is highly encouraging as we move toward clinical translation. These studies come as further validation of the very promising results obtained previously in both nematode and zebrafish models of adult progeria." "These findings deepen our conviction that Telomir-1 can be a first-in-class therapeutic platform for rare aging syndromes and broader age-related diseases," said Erez Aminov, CEO of Telomir. "By demonstrating the ability to reverse cellular damage in human progeria cells, Telomir-1 represents a potential breakthrough for children who currently have no real options beyond modestly delaying the inevitable. This work also lays the foundation for broader applications in neurodegeneration, metabolic dysfunction, and systemic aging. The new data also build on previously reported studies in zebrafish and C. elegans nematodes harboring the wrn gene mutation (a model of adult progeria, or Werner syndrome), where Telomir-1 significantly extended lifespan, restored telomere length, reversed muscle degeneration, and normalized molecular age markers. Telomir is currently finalizing IND-enabling studies for Telomir-1 and plans to engage with the U.S. Food and Drug Administration (FDA) to explore regulatory pathways, including the potential for orphan drug designation. The company is evaluating multiple rare disease indications for initial clinical development. Cautionary Note Regarding Forward-Looking Statements This press release, statements of Telomir's management or advisors related thereto, and the statements contained in the news story linked in this release contain "forward-looking statements," which are statements other than historical facts made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These risks and uncertainties include, but are not limited to, the potential use of the data from our studies, our ability to develop and commercialize Telomir-1 for specific indications, and the safety of Telomir-1. Any forward-looking statements in this press release are based on Telomir's current expectations, estimates and projections only as of the date of this release. These and other risks concerning Telomir's programs and operations are described in additional detail in its Annual Report on Form 10-K for the fiscal year ended December 31, 2024, which are on file with the SEC and available at Telomir explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. Contact Information Helga Moya info@ 396-6723 SOURCE: Telomir Pharmaceuticals, Inc
Indianapolis Star
4 days ago
- Health
- Indianapolis Star
Telomir Pharmaceuticals Prevents Cellular Aging in Patient-Derived Cells from Children with Progeria – an Ultra-Rare Genetic Disorder that Causes Rapid Aging
Study used cell lines obtained from the Progeria Research Foundation to evaluate Telomir-1's effects on key drivers of accelerated aging MIAMI, FLORIDA / ACCESS Newswire Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), a preclinical-stage biotechnology company focused on reversing biological aging and age-related diseases, today announced compelling 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 (HGPS), 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. There are an estimated 400-500 known cases worldwide, including fewer than 30 children currently living with the disease in the United States. Symptoms typically begin within the first two years of life and include growth failure, joint stiffness, loss of body fat and hair, and severe cardiovascular disease. Children with progeria have an average life expectancy of just 13 to 15 years, with most dying from heart attacks or strokes at a young age. The only FDA-approved therapy for progeria, Zokinvy® (lonafarnib), is a farnesyltransferase inhibitor that has been shown to extend lifespan by an average of 4.3 years. However, Zokinvy does not reverse the underlying disease pathology or halt cardiovascular deterioration, which remains the leading cause of death. No approved therapy restores normal cell function or reverses the biological hallmarks of accelerated aging in progeria, highlighting a significant and urgent unmet medical need. 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 (ROS), and intracellular calcium signaling – a marker of mitochondrial dysfunction – under both normal and stress-induced conditions. Key findings include: Improved cell viability: 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. Reduction of oxidative stress: Progeria cells exhibited abnormally high levels of reactive oxygen species (ROS), 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. Restoration of mitochondrial function: 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. 'These results provide the strongest evidence to date that Telomir-1 is not only protective but also restorative at the molecular and cellular level,' said Dr. Angel, Chief Scientific Advisor of Telomir. 'What's especially promising is that the improvements we observed directly target the mechanisms known to drive disease progression in progeria – oxidative stress, metal toxicity, and mitochondrial instability. This level of functional rescue in actual patient-derived cells is highly encouraging as we move toward clinical translation. These studies come as further validation of the very promising results obtained previously in both nematode and zebrafish models of adult progeria.' 'These findings deepen our conviction that Telomir-1 can be a first-in-class therapeutic platform for rare aging syndromes and broader age-related diseases,' said Erez Aminov, CEO of Telomir. 'By demonstrating the ability to reverse cellular damage in human progeria cells, Telomir-1 represents a potential breakthrough for children who currently have no real options beyond modestly delaying the inevitable. This work also lays the foundation for broader applications in neurodegeneration, metabolic dysfunction, and systemic aging. The new data also build on previously reported studies in zebrafish and C. elegans nematodes harboring the wrn gene mutation (a model of adult progeria, or Werner syndrome), where Telomir-1 significantly extended lifespan, restored telomere length, reversed muscle degeneration, and normalized molecular age markers. Telomir is currently finalizing IND-enabling studies for Telomir-1 and plans to engage with the U.S. Food and Drug Administration (FDA) to explore regulatory pathways, including the potential for orphan drug designation. The company is evaluating multiple rare disease indications for initial clinical development. Cautionary Note Regarding Forward-Looking Statements This press release, statements of Telomir's management or advisors related thereto, and the statements contained in the news story linked in this release contain 'forward-looking statements,' which are statements other than historical facts made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These risks and uncertainties include, but are not limited to, the potential use of the data from our studies, our ability to develop and commercialize Telomir-1 for specific indications, and the safety of Telomir-1. Any forward-looking statements in this press release are based on Telomir's current expectations, estimates and projections only as of the date of this release. These and other risks concerning Telomir's programs and operations are described in additional detail in its Annual Report on Form 10-K for the fiscal year ended December 31, 2024, which are on file with the SEC and available at Telomir explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. Contact Information SOURCE: Telomir Pharmaceuticals, Inc View the original press release on ACCESS Newswire
Yahoo
13-06-2025
- Business
- Yahoo
TELO Rises on Promising Telomir-1 Preclinical Results
Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) is among the 10 Best Growth Stocks Under $100 to Buy Now. On Wednesday, Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) revealed that its leading product, Telomir-1, showcased a marked improvement in the neurological, liver, and kidney symptoms observed in a preclinical animal model of Wilson's disease. In a zebrafish model mimicking Wilson's disease, the drug showed substantial dose-dependent improvements, including a decrease in tremors, a normal swimming behavior, and a 50% reduction in copper accumulation in liver tissue. It also led to better liver and kidney histopathology while restoring key biomarkers. As Dr. Angel, Chief Scientific Advisor at Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), mentioned, 'These recent findings establish Telomir-1 as a potent disease-modifying compound in a clinically relevant model of Wilson's disease.' A biopharmaceutical research laboratory filled with scientists, illuminated by the glow of their equipment. With a market capitalization of $59.079 million, the company is also positioning its drug through IND-enabling studies, with plans to submit its first IND for a rare disease by the year's end, followed by human trials in the first half of the next year. Analysts have set a price target of $15.50 for Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), implying an upside of a whopping 688%. These well-structured plans highlight the company's position to lead the global market in the years ahead. Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) is a pre-clinical stage biopharmaceutical company that emphasizes the reversal of oxidative stress, cellular protection, and regulatory mechanisms. Incorporated in 2021, this Florida-based company is designing and developing products to address the causes of rare diseases. While we acknowledge the potential of TELO as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
13-06-2025
- Business
- Yahoo
TELO Rises on Promising Telomir-1 Preclinical Results
Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) is among the 10 Best Growth Stocks Under $100 to Buy Now. On Wednesday, Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) revealed that its leading product, Telomir-1, showcased a marked improvement in the neurological, liver, and kidney symptoms observed in a preclinical animal model of Wilson's disease. In a zebrafish model mimicking Wilson's disease, the drug showed substantial dose-dependent improvements, including a decrease in tremors, a normal swimming behavior, and a 50% reduction in copper accumulation in liver tissue. It also led to better liver and kidney histopathology while restoring key biomarkers. As Dr. Angel, Chief Scientific Advisor at Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), mentioned, 'These recent findings establish Telomir-1 as a potent disease-modifying compound in a clinically relevant model of Wilson's disease.' A biopharmaceutical research laboratory filled with scientists, illuminated by the glow of their equipment. With a market capitalization of $59.079 million, the company is also positioning its drug through IND-enabling studies, with plans to submit its first IND for a rare disease by the year's end, followed by human trials in the first half of the next year. Analysts have set a price target of $15.50 for Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), implying an upside of a whopping 688%. These well-structured plans highlight the company's position to lead the global market in the years ahead. Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) is a pre-clinical stage biopharmaceutical company that emphasizes the reversal of oxidative stress, cellular protection, and regulatory mechanisms. Incorporated in 2021, this Florida-based company is designing and developing products to address the causes of rare diseases. While we acknowledge the potential of TELO as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Indianapolis Star
11-06-2025
- Business
- Indianapolis Star
Telomir Pharmaceuticals Announces Telomir-1 Demonstrates Dose-Dependent Restoration of Neurological, Liver and Kidney Functions in Preclinical Wilson's Disease Model
Treatment reversed tremors, ataxia, anxiety-like behavior, liver and kidney pathology damage, reduced copper accumulation, normalized ALT, AST, and bilirubin levels, and improved survival. Building on previous data in Werner syndrome, showing Telomir-1 reset the epigenetic clock, extended telomere length, restored gene expression, reversed muscle loss, and rescued survival-alongside data in Age Related Macular Degeneration (AMD) demonstrating retinal regeneration and restored vision-the drug continues to show broad regenerative potential across rare genetic degenerative diseases MIAMI, FL / ACCESS Newswire Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), or the 'Company,' a preclinical-stage biotechnology company focused on reversing biological aging and degenerative diseases, today announced compelling new preclinical data demonstrating that its lead drug candidate, Telomir-1, significantly reversed neurological, hepatic and kidney symptoms in a clinically relevant animal model of Wilson's disease. Wilson's disease is a rare and potentially fatal genetic disorder caused by mutations in the ATP7B gene, which impair the body's ability to eliminate excess copper. As copper builds up-primarily in the liver and brain-it leads to inflammation, tissue damage, and multi-organ dysfunction. Patients may experience liver failure, psychiatric disturbances, tremors, and progressive neurological decline. Current treatments involve lifelong copper chelation or liver transplantation, highlighting the urgent need for safer, disease-modifying therapies. These new results build on earlier in vitro studies that confirmed Telomir-1's high binding affinity for copper and its ability to exchange and regulate key ions. The current in vivo findings now confirm that Telomir-1's copper-regulating properties translate into meaningful behavioral, physiological and histological improvements in a genetic animal model of Wilson's disease. In the ATP7B C271X -/- zebrafish model, which mimics human Wilson's disease Telomir-1 demonstrated dose-dependent and statistically significant reversal of major disease features: Key Findings from the Study: Up to a 4- to 5-fold reduction in episodic tremor events Normalization of swim distance, swim velocity, and exploratory behavior Reversal of ataxia-like motor behaviors (e.g., abnormal body bends and turn angles) Approximately 50% reduction in copper accumulation in dry liver tissue Marked improvement in liver and kidney histopathology, with liver and kidney scores reduced to near-normal levels Normalization of ALT, AST, and bilirubin-three critical liver biomarkers. ALT and AST are enzymes elevated during liver injury, while bilirubin builds up when detoxification is impaired. Telomir-1 restored these to wild-type levels, indicating protection of liver function and copper-induced damage Improved survival under high copper exposure conditions 'We've now seen Telomir-1 generate breakthrough results across some of the most challenging age-related and genetic diseases-including AMD, Wilson's disease, cancer, progeroid and Werner syndromes, and early findings suggesting reversal of key factors in type 2 diabetes,' said Erez Aminov, Chairman and CEO of Telomir. 'In our AMD model, Telomir-1 restored vision and regenerated retinal structure using FDA-recognized surrogate endpoints. In Werner syndrome, it reset the epigenetic clock and reversed hallmarks of accelerated aging. And in Wilson's disease, we saw a reversal of neurological, liver and kidney damage. These results point to the broad therapeutic potential of Telomir-1 across diseases driven by cellular degeneration. We believe this positions Telomir-1 as a powerful new platform for longevity, neuroprotection, and regenerative medicine.' 'These recent findings establish Telomir-1 as a potent disease-modifying compound in a clinically relevant model of Wilson's disease,' added Dr. Angel, Chief Scientific Advisor. 'Its ability to reverse behavioral and neurological dysfunction, normalize histological and functional biomarkers, and extend survival underscores its therapeutic promise across both rare and age-related disorders.' Telomir Pharmaceuticals is currently advancing Telomir-1 through IND-enabling studies and expects to file its first IND for a rare disease indication by year-end, with human clinical trials planned for the first half of 2026. Cautionary Note Regarding Forward-Looking Statements This press release, statements of Telomir's management or advisors related thereto, and the statements contained in the news story linked in this release contain 'forward-looking statements,' which are statements other than historical facts made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These risks and uncertainties include, but are not limited to, the potential use of the data from our studies, our ability to develop and commercialize Telomir-1 for specific indications, and the safety of Telomir-1. Any forward-looking statements in this press release are based on Telomir's current expectations, estimates and projections only as of the date of this release. These and other risks concerning Telomir's programs and operations are described in additional detail in its Annual Report on Form 10-K for the fiscal year ended December 31, 2024, which is on file with the SEC. Telomir explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law. Contact Information SOURCE: Telomir Pharmaceuticals, Inc View the original press release on ACCESS Newswire
