Latest news with #WernerSyndrome
Associated Press
09-06-2025
- Health
- Associated Press
Niagen Bioscience Announces First-Ever Peer-Reviewed Study Highlighting the Potential of Nicotinamide Riboside (NR) for Werner Syndrome, a Rare Genetic Disorder
LOS ANGELES--(BUSINESS WIRE)--Jun 9, 2025-- Niagen Bioscience, Inc. (NASDAQ: NAGE) (formerly ChromaDex Corp.), the global authority on NAD+ (nicotinamide adenine dinucleotide) with a focus on the science of healthy aging, shares positive results from a clinical study published in the peer-reviewed journal Aging Cell, by a team led by Masaya Koshizaka, M.D., Ph.D., Associate Professor, and Koutaro Yokote, M.D., Ph.D., MBA, President of Chiba University, both of the Center for Preventive Medical Sciences, Chiba University, and the Department of Diabetes, Metabolism and Endocrinology, Chiba University Hospital, Japan. This is the first study to demonstrate the safety and efficacy of Niagen Bioscience's patented nicotinamide riboside (NR) ingredient, Niagen ®, in individuals with Werner syndrome (WS), a rare genetic disorder marked by rapid aging and premature mortality. The newly published double-blind, placebo-controlled study found that daily supplementation with Niagen significantly elevated blood NAD+ levels by approximately 140% and improved multiple clinical markers of cardiovascular and skin health in individuals with WS. Affecting approximately 1 in 380,000 to 1 in 1,000,000 people globally ( GeneReviews ), Werner syndrome is caused by mutations that impair DNA repair, leading to cellular aging decades ahead of normal progression. Dr. Koshizaka noted, 'We hope our work will accelerate studies on not only WS but also other premature aging disorders and common age-related diseases—ultimately helping to extend health span and improve quality of life in both patients and the broader population.' Developing NAD+ Therapies for Rare, Age-Related Diseases This study builds on the growing body of clinical research demonstrating Niagen's potential in rare, age-related diseases (see Table 1 below). In Ataxia Telangiectasia (AT), Niagen has shown improvements in neurological function, coordination, and immune markers, including in pediatric populations. Rob Fried, CEO of Niagen Bioscience, stated, 'Rare disease research is a priority for Niagen Bioscience, particularly those indications associated with accelerated aging, mitochondrial dysfunction, or NAD+ deficiency.' The U.S. Food and Drug Administration (FDA) previously granted Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) Designation to NR for the treatment of AT. These designations underscore the urgent unmet need and potential therapeutic value of Niagen in rare disease populations. Vilhelm Bohr, M.D., Ph.D., former National Institute on Aging (NIA) Chief of the Laboratory of Molecular Genetics, and current Affiliate Professor in Genome Instability and Neurodegeneration at the University of Copenhagen and Scientific Advisor to Niagen Bioscience and coauthor on the study, commented, 'Werner syndrome is a rare genetic disorder that has long served as a valuable model for understanding the mechanisms of human aging. This study marks the first clinical trial using Niagen in those with Werner syndrome, and the findings—particularly improvements in cardiovascular markers—are promising. As Werner syndrome is a well-established model for normal aging, these results also suggest that NAD+ supplementation may support healthier aging in the broader population.' Study Highlights This 52-week randomized, double-blind, placebo-controlled crossover trial evaluated the safety and efficacy of oral Niagen supplementation in individuals with WS. The study randomized nine individuals (mean age: 47) who received 1,000 mg/day of Niagen or placebo for 26 weeks before crossing over to the alternate treatment for an additional 26 weeks. Key findings include: This study represents the first clinical evaluation of Niagen in WS and supports further investigation of NAD+ augmentation as a therapeutic strategy in rare progeroid diseases. For additional information on the science supporting Niagen ®, visit About Niagen Bioscience: Niagen Bioscience, Inc. (NASDAQ: NAGE), formerly ChromaDex Corp., is the global leader in NAD+ (nicotinamide adenine dinucleotide) science and healthy-aging research. As a trusted pioneer of NAD+ discoveries, Niagen Bioscience™ is dedicated to advancing healthspan through precision science and innovative NAD+-boosting solutions. The Niagen Bioscience team, composed of world-renowned scientists, works with independent investigators from esteemed universities and research institutions around the globe to uncover the full potential of NAD+. A vital coenzyme found in every cell of the human body, NAD+ declines with age and exposure to everyday lifestyle stressors. NAD+ depletion is a key contributor to age-related changes in health and vitality. Distinguished by state-of-the-art laboratories, rigorous scientific and quality protocols, and collaborations with leading research institutions worldwide, Niagen Bioscience sets the gold standard for research, quality, and innovation. There's a better way to age. At the heart of its clinically proven product portfolio is Niagen ® (patented nicotinamide riboside, or NR), the most efficient, well-researched, high-quality, and legal NAD+ booster available. Niagen Bioscience's robust patent portfolio protects NR and other NAD+ precursors. Niagen Bioscience maintains a website at where copies of press releases, news, and financial information are regularly published. Forward Looking Statements: This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended, including statements related to infringement or non-infringement of intellectual property rights. Statements that are not a description of historical facts constitute forward-looking statements and may often, but not always, be identified by the use of such words as 'expects,' 'anticipates,' 'intends,' 'estimates,' 'plans,' 'potential,' 'possible,' 'probable,' 'believes,' 'seeks,' 'may,' 'will,' 'should,' 'could' or the negative of such terms or other similar expressions. Risks that contribute to the uncertain nature of these forward-looking statements include the impact of the COVID-19 pandemic on our business and the global economy; our history of operating losses and need to obtain additional financing; the growth and profitability of our product sales; our ability to maintain sales, marketing and distribution capabilities; changing consumer perceptions of our products; our reliance on a single or limited number of third-party suppliers; and the risks and uncertainties associated with our business and financial condition. Xx Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and actual results may differ materially from those suggested by these forward-looking statements. All forward-looking statements are qualified in their entirety by this cautionary statement and Niagen Bioscience undertakes no obligation to revise or update this release to reflect events or circumstances after the date hereof. View source version on CONTACT: Niagen Bioscience Media Contact: Kendall Knysch, Senior Director of Media Relations & Partnerships 310.405.5227 [email protected] Bioscience Investor Relations Contact: ICR, LLC Reed Anderson (646) 277-1260 Stephanie Carrington (646) 277-1282 [email protected] KEYWORD: NORTH AMERICA UNITED STATES ASIA PACIFIC EUROPE JAPAN CALIFORNIA INDUSTRY KEYWORD: RESEARCH VITAMINS/SUPPLEMENTS GENETICS CLINICAL TRIALS BIOTECHNOLOGY ALTERNATIVE MEDICINE HEALTH PHARMACEUTICAL SCIENCE SOURCE: Niagen Bioscience, Inc Copyright Business Wire 2025. PUB: 06/09/2025 08:32 AM/DISC: 06/09/2025 08:31 AM
Yahoo
09-06-2025
- Health
- Yahoo
Niagen Bioscience Announces First-Ever Peer-Reviewed Study Highlighting the Potential of Nicotinamide Riboside (NR) for Werner Syndrome, a Rare Genetic Disorder
Results demonstrated that nicotinamide riboside (NR) significantly elevated NAD+ levels and improved multiple clinical markers in people with Werner Syndrome Niagen Bioscience expands rare disease research portfolio, supporting further investigation of NAD+ augmentation with NR as a therapeutic strategy in rare progeroid diseases LOS ANGELES, June 09, 2025--(BUSINESS WIRE)--Niagen Bioscience, Inc. (NASDAQ: NAGE) (formerly ChromaDex Corp.), the global authority on NAD+ (nicotinamide adenine dinucleotide) with a focus on the science of healthy aging, shares positive results from a clinical study published in the peer-reviewed journal Aging Cell, by a team led by Masaya Koshizaka, M.D., Ph.D., Associate Professor, and Koutaro Yokote, M.D., Ph.D., MBA, President of Chiba University, both of the Center for Preventive Medical Sciences, Chiba University, and the Department of Diabetes, Metabolism and Endocrinology, Chiba University Hospital, Japan. This is the first study to demonstrate the safety and efficacy of Niagen Bioscience's patented nicotinamide riboside (NR) ingredient, Niagen®, in individuals with Werner syndrome (WS), a rare genetic disorder marked by rapid aging and premature mortality. The newly published double-blind, placebo-controlled study found that daily supplementation with Niagen significantly elevated blood NAD+ levels by approximately 140% and improved multiple clinical markers of cardiovascular and skin health in individuals with WS. Affecting approximately 1 in 380,000 to 1 in 1,000,000 people globally (GeneReviews), Werner syndrome is caused by mutations that impair DNA repair, leading to cellular aging decades ahead of normal progression. Dr. Koshizaka noted, "We hope our work will accelerate studies on not only WS but also other premature aging disorders and common age-related diseases—ultimately helping to extend health span and improve quality of life in both patients and the broader population." Developing NAD+ Therapies for Rare, Age-Related Diseases This study builds on the growing body of clinical research demonstrating Niagen's potential in rare, age-related diseases (see Table 1 below). In Ataxia Telangiectasia (AT), Niagen has shown improvements in neurological function, coordination, and immune markers, including in pediatric populations. Rob Fried, CEO of Niagen Bioscience, stated, "Rare disease research is a priority for Niagen Bioscience, particularly those indications associated with accelerated aging, mitochondrial dysfunction, or NAD+ deficiency." The U.S. Food and Drug Administration (FDA) previously granted Orphan Drug Designation (ODD) and Rare Pediatric Disease (RPD) Designation to NR for the treatment of AT. These designations underscore the urgent unmet need and potential therapeutic value of Niagen in rare disease populations. Why NAD+ Matters in Rare DiseaseScientific research has shown that declining NAD+ levels can contribute to age-related decline, mitochondrial dysfunction, and impaired DNA repair. In both Werner syndrome and AT, NAD+ deficiency appears to play a central role in disease progression (Fang et al., 2019). By restoring NAD+ levels, Niagen may help activate protective enzymes like SIRT1 and PARP1, reduce oxidative stress, and improve tissue function (Fang et al., 2016; Veenhuis et al., 2021; Presterud et al., 2023; Shoji et al., 2025; Lautrup et al., 2025). Vilhelm Bohr, M.D., Ph.D., former National Institute on Aging (NIA) Chief of the Laboratory of Molecular Genetics, and current Affiliate Professor in Genome Instability and Neurodegeneration at the University of Copenhagen and Scientific Advisor to Niagen Bioscience and coauthor on the study, commented, "Werner syndrome is a rare genetic disorder that has long served as a valuable model for understanding the mechanisms of human aging. This study marks the first clinical trial using Niagen in those with Werner syndrome, and the findings—particularly improvements in cardiovascular markers—are promising. As Werner syndrome is a well-established model for normal aging, these results also suggest that NAD+ supplementation may support healthier aging in the broader population." Study Highlights This 52-week randomized, double-blind, placebo-controlled crossover trial evaluated the safety and efficacy of oral Niagen supplementation in individuals with WS. The study randomized nine individuals (mean age: 47) who received 1,000 mg/day of Niagen or placebo for 26 weeks before crossing over to the alternate treatment for an additional 26 weeks. Key findings include: Robust increase in NAD+ levels: Niagen supplementation led to a ~140% increase in plasma NAD+ levels, compared to a ~4% decrease in the placebo group. Improved arterial stiffness: Niagen significantly improved cardio-ankle vascular index (CAVI), a measure of arterial stiffness. Cardioprotective lipid shift: Niagen increased the number of large HDL particles, indicating potential cardiovascular benefits. Wound healing support: Niagen reduced skin ulcer size and heel pad thinning, while ulcers worsened in the placebo group. Clinical safety profile: No moderate or severe adverse events were reported. Mild adverse events were fewer in individuals who received Niagen (7) compared to those who received placebo (12). Tolerability in complex individuals: Although mild liver enzyme elevations were noted, they were deemed manageable and consistent with underlying liver sensitivities common in Werner syndrome. This study represents the first clinical evaluation of Niagen in WS and supports further investigation of NAD+ augmentation as a therapeutic strategy in rare progeroid diseases. For additional information on the science supporting Niagen®, visit Table 1The table below provides an overview of the clinical research published to date on Niagen in rare age-related conditions. Publication Dose Duration Health Area Study Design Key Outcomes Shoji et al., 2025 1000 mg 52 weeks Werner syndrome Randomized, double-blind, placebo-controlled crossover study in 11 individuals with Werner syndrome ~140% increase in plasma NAD+ levels; improved arterial stiffness and reduced skin ulcer size. No moderate or severe adverse events reported. Presterud et al., 2023 500 mg 2 years Ataxia Telangiectasia (AT) Open-label, single-arm observational study in 10 AT individuals Significant improvements in motor coordination and eye movements. No serious adverse events. Longest NR supplementation trial to date. Veenhuis et al., 2021 25 mg/kg 4 months Ataxia Telangiectasia (AT) Open-label proof-of-concept study in 24 AT individuals Improved ataxia scores (SARA, ICARS); effects reversed after withdrawal. Increased IgG levels in immunodeficient individuals. Tinnevelt et al., 2020 25 mg/kg 4 months Ataxia Telangiectasia (AT) Comparative study in 14 individuals with AT NR-related pathways and metabolites significantly increased following NR supplementation. About Niagen Bioscience: Niagen Bioscience, Inc. (NASDAQ: NAGE), formerly ChromaDex Corp., is the global leader in NAD+ (nicotinamide adenine dinucleotide) science and healthy-aging research. As a trusted pioneer of NAD+ discoveries, Niagen Bioscience™ is dedicated to advancing healthspan through precision science and innovative NAD+-boosting solutions. The Niagen Bioscience team, composed of world-renowned scientists, works with independent investigators from esteemed universities and research institutions around the globe to uncover the full potential of NAD+. A vital coenzyme found in every cell of the human body, NAD+ declines with age and exposure to everyday lifestyle stressors. NAD+ depletion is a key contributor to age-related changes in health and vitality. Distinguished by state-of-the-art laboratories, rigorous scientific and quality protocols, and collaborations with leading research institutions worldwide, Niagen Bioscience sets the gold standard for research, quality, and innovation. There's a better way to age. At the heart of its clinically proven product portfolio is Niagen® (patented nicotinamide riboside, or NR), the most efficient, well-researched, high-quality, and legal NAD+ booster available. Niagen Bioscience's robust patent portfolio protects NR and other NAD+ precursors. Niagen Bioscience maintains a website at where copies of press releases, news, and financial information are regularly published. Forward Looking Statements: This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended, including statements related to infringement or non-infringement of intellectual property rights. Statements that are not a description of historical facts constitute forward-looking statements and may often, but not always, be identified by the use of such words as "expects," "anticipates," "intends," "estimates," "plans," "potential," "possible," "probable," "believes," "seeks," "may," "will," "should," "could" or the negative of such terms or other similar expressions. Risks that contribute to the uncertain nature of these forward-looking statements include the impact of the COVID-19 pandemic on our business and the global economy; our history of operating losses and need to obtain additional financing; the growth and profitability of our product sales; our ability to maintain sales, marketing and distribution capabilities; changing consumer perceptions of our products; our reliance on a single or limited number of third-party suppliers; and the risks and uncertainties associated with our business and financial condition. Xx Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and actual results may differ materially from those suggested by these forward-looking statements. All forward-looking statements are qualified in their entirety by this cautionary statement and Niagen Bioscience undertakes no obligation to revise or update this release to reflect events or circumstances after the date hereof. View source version on Contacts Niagen Bioscience Media Contact: Kendall Knysch, Senior Director of Media Relations & Niagen Bioscience Investor Relations Contact: ICR, LLCReed Anderson(646) 277-1260Stephanie Carrington(646) 277-1282niagenir@
Miami Herald
05-06-2025
- Health
- Miami Herald
Telomir Pharmaceuticals Announces Telomir-1 Resets the Body's Epigenetic Clock, Reverses DNA Methylation, and Restores Youthful Gene Regulation in an Ultra-Rare Accelerated Aging Animal Model of Werner Syndrome
Treatment restored gene control, extended telomere length beyond healthy levels, reversed muscle and weight loss, reduced oxidative stress, and resulted in 100% survival - highlighting Telomir-1's potential to reverse key hallmarks of aging MIAMI, FL / ACCESS Newswire / June 5, 2025 / Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), or the "Company," an emerging leader in age-reversal science, today announced compelling 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 (WS)-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. Werner Syndrome: A Rare and Devastating Accelerated Aging Disorder Werner Syndrome is a rare autosomal recessive disorder caused by mutations in the wrn gene, which plays a critical role in DNA repair and telomere maintenance. Patients typically begin showing signs of premature aging-such as graying hair, cataracts, diabetes, osteoporosis, and atherosclerosis-in their 20s or 30s. Median life expectancy is 40-50 years. There are currently no FDA-approved treatments. To model this disease, the Company's study utilized the Sen57wrn-/-ND6-/+ zebrafish model, which replicates key features of Werner Syndrome, including telomere loss, mitochondrial dysfunction, retinal degeneration, muscle deterioration, and abnormal DNA methylation. Key Preclinical Findings 1. DNA Methylation Reversal - Resetting the Genetic ClockDNA 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 (called methyl groups) 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 (via silencing of tumor suppressor genes)Neurodegenerative diseases like Alzheimer's and Parkinson'sAutoimmune diseases such as lupus and multiple sclerosisMetabolic conditions like Type 2 diabetesPremature 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. 2. Telomere Elongation - Rebuilding the Chromosomal ClockTelomeres 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 (healthy) levels, suggesting not only restoration but also potential enhancement of chromosomal integrity 3. Muscle Mass and Body Weight RecoveryIn 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. 4. Oxidative Stress ReductionReactive oxygen species (ROS), which damage cells and accelerate aging, were elevated in untreated animals. Telomir-1 reduced ROS levels by up to 50%, suggesting improved cellular resilience. 5. Survival BenefitRoughly 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. "These results reinforce my conviction that Telomir-1 may represent one of the most important scientific developments in the field of aging," said Erez Aminov, Chairman and CEO of Telomir. "While we remain in the preclinical phase, the consistency and strength of the data demand serious attention. My full commitment is behind advancing Telomir-1 toward human studies in the most responsible and rigorous way. The potential here is too significant to ignore." "This is one of the most comprehensive rejuvenation profiles we've seen in a vertebrate aging model," added Dr. Itzchak Angel, Chief Scientific Advisor. "The ability to reverse both genomic and epigenetic instability while improving survival supports the growing potential of Telomir-1 as a foundational therapy for aging-related diseases." 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 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 safety of Telomir-1. 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 Helga Moya info@ 396-6723 SOURCE: Telomir Pharmaceuticals, Inc
Indianapolis Star
05-06-2025
- Business
- Indianapolis Star
Telomir Pharmaceuticals Announces Telomir-1 Resets the Body's Epigenetic Clock, Reverses DNA Methylation, and Restores Youthful Gene Regulation in an Ultra-Rare Accelerated Aging Animal Model of Werner Syndrome
Treatment restored gene control, extended telomere length beyond healthy levels, reversed muscle and weight loss, reduced oxidative stress, and resulted in 100% survival – highlighting Telomir-1's potential to reverse key hallmarks of aging MIAMI, FL / ACCESS Newswire Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), or the 'Company,' an emerging leader in age-reversal science, today announced compelling 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 (WS) -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. Werner Syndrome: A Rare and Devastating Accelerated Aging Disorder Werner Syndrome is a rare autosomal recessive disorder caused by mutations in the wrn gene, which plays a critical role in DNA repair and telomere maintenance. Patients typically begin showing signs of premature aging-such as graying hair, cataracts, diabetes, osteoporosis, and atherosclerosis-in their 20s or 30s. Median life expectancy is 40-50 years. There are currently no FDA-approved treatments. To model this disease, the Company's study utilized the Sen57wrn-/-ND6-/+ zebrafish model, which replicates key features of Werner Syndrome, including telomere loss, mitochondrial dysfunction, retinal degeneration, muscle deterioration, and abnormal DNA methylation. Key Preclinical Findings 1. DNA Methylation Reversal – Resetting the Genetic Clock 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 (called methyl groups) 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 (via silencing of tumor suppressor genes) 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. 2. Telomere Elongation – Rebuilding the Chromosomal Clock 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 (healthy) levels, suggesting not only restoration but also potential enhancement of chromosomal integrity 3. Muscle Mass and Body Weight Recovery 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. 4. Oxidative Stress Reduction Reactive oxygen species (ROS), which damage cells and accelerate aging, were elevated in untreated animals. Telomir-1 reduced ROS levels by up to 50%, suggesting improved cellular resilience. 5. Survival Benefit 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. 'These results reinforce my conviction that Telomir-1 may represent one of the most important scientific developments in the field of aging,' said Erez Aminov, Chairman and CEO of Telomir. 'While we remain in the preclinical phase, the consistency and strength of the data demand serious attention. My full commitment is behind advancing Telomir-1 toward human studies in the most responsible and rigorous way. The potential here is too significant to ignore.' 'This is one of the most comprehensive rejuvenation profiles we've seen in a vertebrate aging model,' added Dr. Itzchak Angel, Chief Scientific Advisor. 'The ability to reverse both genomic and epigenetic instability while improving survival supports the growing potential of Telomir-1 as a foundational therapy for aging-related diseases.' 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 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 safety of Telomir-1. 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
Yahoo
05-06-2025
- Business
- Yahoo
EXCLUSIVE: Telomir Pharmaceuticals Lead Drug Shows Promising Signs In Animal Study For Rare Premature Aging Disorder
Telomir Pharmaceuticals Inc. (NASDAQ:TELO) on Thursday released new preclinical data for its lead candidate, Telomir-1. The data demonstrated that 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 (WS)—a rare genetic disorder also known as adult-onset progeria. Adult progeria is a rare genetic disorder characterized by premature aging starting in late adolescence or early Syndrome is caused by mutations in the wrn gene, which plays a critical role in DNA repair and telomere maintenance. 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. 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, Telomir-1 increased telomere length by about three-fold compared to the shortened length in the mutated animals. At the higher dose, telomere length significantly exceeded wild-type (healthy) levels, suggesting restoration and 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 (ROS), 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. In March, Telomir Pharmaceuticals revealed preclinical findings demonstrating that Telomir-1 reduces tumor size by approximately 50% in a prostate cancer animal model with aggressive cancer cells. The company says that Telomir-1 actively suppresses cancer growth and protects against chemotherapy-induced toxicity and mortality when combined with Paclitaxel—a widely used chemotherapy but often associated with severe toxicity and side effects. Price Action: TELO stock closed at $2.11 on Wednesday. Read Next:Up Next: Transform your trading with Benzinga Edge's one-of-a-kind market trade ideas and tools. Click now to access unique insights that can set you ahead in today's competitive market. Get the latest stock analysis from Benzinga? This article EXCLUSIVE: Telomir Pharmaceuticals Lead Drug Shows Promising Signs In Animal Study For Rare Premature Aging Disorder originally appeared on © 2025 Benzinga does not provide investment advice. All rights reserved. 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
