Latest news with #MolecularTherapy
Yahoo
25-06-2025
- Health
- Yahoo
Protein Breakthrough Could Extend Healthy Lifespan, Study Finds
A protein called Klotho, when boosted, helps mice live longer and age healthier, offering a potential new path for anti-aging treatments in humans, according to a groundbreaking study from the Universitat Autònoma de Barcelona (UAB). Published in Molecular Therapy and led by Professor Miguel Chillón, the study found that a specific form of Klotho, known as secreted Klotho (s-KL), improved muscle strength, bone health, and brain function in treated mice. These mice lived 15–20% longer than untreated peers, reaching the human equivalent of about 70 years with greater physical and mental vitality. Klotho is known to combat inflammation and oxidative stress, key drivers of aging. Unlike other forms of the protein, s-KL circulates freely in the body without disrupting systems like calcium metabolism, making it a safer candidate for therapies. 'If we can find a viable delivery method, s-KL could make a significant contribution to improving people's quality of life,' the researchers said, per The Brighter Side of News. 'It could help build the healthiest society possible.' Using gene therapy with adeno-associated viruses (AAV9), scientists delivered s-KL through the bloodstream and directly into the brain, targeting areas critical for muscle and memory. Three groups of mice were treated: one at six months (young adulthood), another at 12 months (middle age), and a control group. By 24 months, treated mice showed stronger muscles with larger fibers, less fibrosis, and enhanced repair by satellite cells. Bone structure, particularly in females, remained more intact, suggesting protection against osteoporosis. 'KL treatment improved physical fitness, related to a reduction in muscle fibrosis and an increase in muscular regenerative capacity,' the researchers noted, The Brighter Side reported. In the brain, s-KL promoted new neuron growth in the hippocampus, the memory center, and boosted immune processes like phagocytosis, where cells clear toxic waste. This cleanup is vital as aging glial cells often fail to remove debris, contributing to cognitive decline. Transcriptomic analysis revealed increased gene activity tied to immune response and brain cell regeneration. 'We now have viral vectors that can reach the brain after being administered intravenously, which would make it easier to safely transfer this therapy to humans,' said Joan Roig-Soriano, the study's first author, per The Brighter Side. The findings aim to extend health span — the years spent in good health — rather than just lifespan. The team has filed patents for using Klotho to support cognition, strengthen bones and muscles, and increase longevity. While gene therapy was used in the study, direct protein delivery as a drug is also being explored, though a reliable method to target tissues without side effects is still needed. With global populations aging — experts predict a quarter of people in developed countries will be over 65 by 2060 — such treatments could ease the burden on health systems by preventing age-related diseases before they start. The study's results suggest Klotho could redefine aging, offering a future where decline is not inevitable.
Yahoo
09-06-2025
- Health
- Yahoo
KLOTHO NEUROSCIENCE, INC. ANNOUNCES AN APPROACH TO INCREASE LONGEVITY AND HEALTHY LIFE SPAN - REPLACE A SILENCED GENE CALLED ALPHA-KLOTHO ("α-KLOTHO")
Recent clinical and pre-clinical studies and analysis indicates the potential to increase lifespan and reduce age-associated degeneration in multiple organ systems has be realized with a focus on the human gene called Klotho. NEW YORK, June 9, 2025 /PRNewswire/ -- Klotho Neurosciences, Inc. (NASDAQ: KLTO), a U.S.-based biogenetics company announced the findings of pre-clinical studies indicating the potential of elevating Klotho gene expression to simultaneously reduce the age-associated degeneration in multiple organs, increasing both life and health span. Pioneering discoveries by Professor Makoto Kuro-O in 1997 showed that Klotho concentrations in the blood were directly associated with lifespan of mammals – the lower the Klotho blood levels the shorter the lifespan. Kuro-O's laboratory then published the first evidence that genetic over expression of the full-length form of Klotho in mice, increased in lifespan of mice of up to 30%-40% longer compared to the normal mouse lifespan. Since then, the Klotho protein has gained much attention because of its ability to influence key biological pathways involved in metabolism, inflammation and tissue repair, which are closely linked to the aging process. A series of experiments led by Joan Roig-Soriano and colleagues, published in the February 2025 edition of Molecular Therapy, highlights the promising role of the naturally occurring secreted form of the Klotho protein ("s-KL") and its effects on healthy aging mice and mice with a rapidly aging phenotype. Key observations were that, while aging is a major risk factor for many pathologies, including cognitive decline, neuroinflammation, sarcopenia, and osteoporosis, the secreted protein s-KL has emerged as a potentially promising therapeutic anti-aging molecule due to its many biological effects involving multiple pathways related to cell injury, stress, and inflammation. The s-KL was administered using an adeno-associated virus serotype 9 delivery vector (AAV9) that expressed the secreted KL protein isoform and efficiently increased the concentration of s-KL in serum, resulting in a 20% increase in lifespan. Dr. Joseph Sinkule, the CEO of Klotho Neurosciences (KLTO) commented that "KLTO has secured an exclusive worldwide license for s-Kl from Universitat Autònoma de Barcelona (UAB) and Institució Catalana de Recerca i Estudis Avançats (ICREA) in Spain. As a result, we have the exclusive use of patents issued in the USA, Europe, and China covering a secreted splice variant of mammalian Klotho referred to as s-KL, as a treatment for neurodegenerative and age -related disorders. Professor Makoto Kuro-O is also a scientific advisor to KLTO. This recently published paper provides further credence to our development of s-KL as a treatment to reduce age-associated degeneration where, as a company, KLTO has a particular focus on neurodegenerative diseases such as ALS, Alzheimer's and Parkinson's disease. The results disclosed in the paper show the potential of elevating s-KL protein expression, resulting in the reduction of age-associated degeneration in multiple organs, increasing both life and health span". About Klotho Neurosciences, Inc. Klotho Neurosciences, Inc. (NASDAQ: KLTO), is a biogenetics company focused on the development of innovative, disease-modifying cell and gene therapies using a protein derived from a patented form of the "anti-aging" human Klotho gene (s-KL), and it's novel delivery systems to transform and improve the treatment of neurodegenerative and age-related disorders such as ALS, Alzheimer's, and Parkinson's disease. The company's current portfolio consists of its proprietary cell and gene therapy programs using DNA and RNA as therapeutics and genomics-based diagnostic assays. The company is managed by a team of individuals and advisors who are highly experienced in biopharmaceutical product development and commercialization. For more information, contact:Investor Contact and Corporate Communications - Jeffrey LeBlanc, CFOjeff@ Website: Forward-Looking Statements: This press release contains forward-looking statements. These statements are made under the "safe harbor" provisions of the U.S. Private Securities Litigation Reform Act of 1995. These forward-looking statements generally are identified by the words "believe," "project," "expect," "anticipate," "estimate," "intend," "strategy," "future," "opportunity," "plan," "may," "should," "will," "would," "will be," "will continue," "will likely result," and similar expressions. Without limiting the generality of the foregoing, the forward-looking statements in this press release include descriptions of the Company's future commercial operations. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. Many factors could cause actual future events to differ materially from the forward-looking statements in this press release, such as the Company's inability to implement its business plans, identify and realize additional opportunities, or meet or exceed its financial projections and changes in the regulatory or competitive environment in which the Company operates. You should carefully consider the foregoing factors and the other risks and uncertainties described in the documents filed or to be filed by the Company with the U.S. Securities and Exchange Commission (the "SEC") from time to time, which could cause actual events and results to differ materially from those contained in the forward-looking statements. Copies of these documents are available on the SEC's website, All information provided herein is as of the date of this press release, and the Company undertakes no obligation to update any forward-looking statement, except as required under applicable law. View original content to download multimedia: SOURCE Klotho Neurosciences, Inc.
The Hindu
22-05-2025
- Health
- The Hindu
CAR-T therapy can be safely manufactured at hospital, finds ICMR-funded trial led by CMC Vellore
An ICMR-funded trial led by CMC Vellore demonstrated that CAR-T therapy, which uses a patient's own T cells to fight cancer, can be safely manufactured at the hospital and infused to treat patients in India at a low cost. For the first time, these CAR-T cells were produced and infused in a hospital in India. CMC Vellore director and principal author of the study Vikram Mathews explained that Chimeric antigen receptor T cells (CAR-T cells) are normal T-cells that are part of the patient's own immune system. In CART-T cell therapy, these cells are engineered to recognise and target the specific cancer cell, thus using the immune system to fight the disease. CAR-T cell therapy has been proven to be very effective even in patients who have failed all other therapies, he said. This process usually involves inserting into the normal T cells, the required genetic information to produce an antibody receptor that will recognise the antigen/substance on the surface of the cancer cell. "This process is usually done in large centralised commercial corporations, which contributes to logistic challenges, increased costs, and decreased efficacy," Dr. Mathews said. One of many strategies to reduce the cost of this therapy, is to produce the CAR-T cells at the hospital site itself, this strategy is called decentralized or point-of-care manufacturing (PoC). In this study, the authors provided evidence that this is feasible in India. What did the study find? Early data from this study establishes its safety and also shows promising results, said Dr Mathews said. A total of 10 patients, aged 6-59 years, six of them with acute leukemia and four with lymphoma, who had failed all earlier treatments, were treated with CAR-T cells manufactured at CMC Vellore under this PoC strategy. The study found that the therapy brought about 100 per cent remission in acute lymphoblastic leukaemia patients, 50 per cent remission in large B-cell lymphoma patients. Overall, eight of the ten patients remained cancer-free at a median follow-up of 15 months since starting the therapy. The doctors further reported that this was safe, well-tolerated, and had minimal side effects. The study, 'Safety, efficacy and total cost of point-of-care manufactured anti-CD19 CAR-T cell therapy in India: VELCART trial' has been published in the journal Molecular Therapy. The study also stated that when CART-T cells are manufactured in this PoC model, the cost of the therapy is nearly 90 per cent less than the global average. The production time for the CART-T cells was nine days in in-hospital settings. Dr Mathews said that the use of a fresh, unfrozen product also contributed to better outcomes. "This trial redefines how cancer therapy can be delivered - efficiently, affordably, and close to patients. India is leading the way in developing next-generation, in-house biotherapies with global relevance," he said. "This model could be easily replicated in most tertiary healthcare facilities in the country," Dr Mathews said.
Yahoo
15-05-2025
- Business
- Yahoo
Voyager Demonstrates ALPL Receptor-Mediated Blood-Brain Barrier Transport of Novel AAV Capsids in Molecular Therapy Publication
LEXINGTON, Mass., May 15, 2025 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, today announced the first peer-reviewed publication of data demonstrating the ability of alkaline phosphatase (ALPL) to transport a novel AAV capsid across the blood-brain barrier (BBB). The article, titled 'Highly conserved brain vascular receptor ALPL mediates transport of engineered AAV vectors across the blood-brain barrier,' was published in Molecular Therapy and can be accessed here. 'Understanding ALPL and its ability to mediate transport across the blood-brain barrier has been foundational to the evolution of our gene therapy programs, two of which are advancing towards IND filings this year with a partner,' said Mathieu Nonnenmacher, Ph.D., Vice President of Gene Therapy at Voyager. 'Building on our first-generation capsids, such as VCAP-102, which is featured in this paper, we have evolved next-generation capsids with even stronger brain transduction and liver de-targeting, as well as stealth capsids with immune-evading capabilities.' The Molecular Therapy paper outlines the generation of novel, cross-species AAV capsid VCAP-102, which demonstrates 20- to 400-fold increased gene transfer across multiple brain regions relative to AAV9 in both rodents and non-human primates (NHP), and the identification of ALPL as the primary receptor used by VCAP-102 to cross the BBB. In addition, the confirmation that the ALPL capsid family binds and demonstrates transcytosis with human ALPL in a cell barrier in vitro model suggests clinical translatability. As previously announced, Voyager presented next-generation and stealth-capsid data at the American Society of Gene & Cell Therapy's (ASGCT) 28th annual meeting. In multiple NHP studies utilizing a variety of payloads, a single intravenous 3e13 vg/kg dose of Voyager's second-generation CNS capsids transduced up to 98% of dopaminergic neurons in substantia nigra, up to 94% of motor neurons in the spinal cord, up to 66% of neurons in the thalamus, up to 43% of neurons in the motor cortex, and 87-99% of astrocytes broadly across brain regions. 'In addition to speeding the evolution of novel capsid families, we are leveraging our work with ALPL and other receptors to deliver diverse classes of non-viral candidates into the CNS,' said Todd Carter, Ph.D., Chief Scientific Officer of Voyager Therapeutics. 'We believe this multi-modality approach, encompassing both viral and non-viral CNS delivery, is critical to addressing unmet needs in neurological disease.' About the TRACER™ Capsid Discovery PlatformVoyager's TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of novel AAV capsids to enable gene therapy. Voyager has leveraged TRACER to create multiple families of novel capsids that, following intravenous delivery in preclinical studies, harness the extensive vasculature of the central nervous system (CNS) to cross the blood-brain barrier and transduce a broad range of CNS regions and cell types. In cross-species preclinical studies (rodents and multiple non-human primate species), intravenous delivery of TRACER-generated capsids resulted in widespread payload expression across the CNS at relatively low doses, enabling selection of multiple development candidates in Voyager's wholly-owned and partnered gene therapy programs for neurologic diseases. About Voyager TherapeuticsVoyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and multiple other diseases of the central nervous system. Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; and Neurocrine Biosciences, Inc. For more information, visit Voyager Therapeutics® is a registered trademark, and TRACER™ is a trademark, of Voyager Therapeutics, Inc. Forward-Looking StatementsThis press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as 'will,' 'anticipated,' 'expect,' 'believe,' 'anticipate,' 'potential,' 'may,' or 'continue,' and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding Voyager's ability to advance its AAV-based gene therapy programs and non-viral CNS delivery programs, including the potential for Voyager's novel TRACER capsids to achieve desired results in humans, including neuronal and glial transduction across multiple brain regions, and ALPL-mediated transcytosis similar to the results demonstrated in rodents and NHPs; potential clinical translatability in humans; increased patient eligibility to receive AAV gene therapies; and expectations for advancement of gene therapy product candidates under the collaboration programs, including anticipated submission of IND filings and initiation of clinical trials in two partnered programs are forward looking. All forward-looking statements are based on estimates and assumptions by Voyager's management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain and subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the continued development of Voyager's technology platforms, including Voyager's TRACER platform and its non-viral discovery platform; Voyager's scientific approach and program development progress, and the restricted supply and increased costs of critical research components; the development by third parties of capsid identification platforms that may be competitive to Voyager's TRACER capsid discovery platform; Voyager's ability to create and protect intellectual property rights associated with the TRACER capsid discovery platform, the capsids identified by the platform, and development candidates for Voyager's pipeline programs; the timing, initiation, conduct and outcomes of Voyager's preclinical and clinical studies; the availability of data from clinical trials; the expectations and decisions of regulatory authorities; the availability or commercial potential of product candidates under collaborations; the success of Voyager's product candidates; the willingness and ability of Voyager's collaboration partners to meet obligations under collaboration agreements with Voyager; the possibility or the timing of Voyager's receipt of program reimbursement, development or commercialization milestones, option exercise, and other payments under Voyager's existing licensing or collaboration agreements; the ability of Voyager to negotiate and complete licensing or collaboration agreements with other parties on terms acceptable to Voyager and the third parties; the success of programs controlled by third-party collaboration partners in which Voyager retains a financial interest; the ability to attract and retain talented directors, employees, and contractors; and the sufficiency of Voyager's cash resources to fund its operations and pursue its corporate objectives. These statements are also subject to a number of material risks and uncertainties that are described in Voyager's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. ContactsTrista Morrison, tmorrison@ Investors: Sarah McCabe, smccabe@ Brooke Shenkin, brooke@ 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
Daily Mail
08-05-2025
- Health
- Daily Mail
Jab duo could extend life by up to 16 years and de-age the brain - but are you brave enough to take it?
Scientists are a step closer to creating a 'fountain of youth' treatment that could help humans live into their nineties—and beyond. The injections involve the 'anti-ageing molecule' klotho, a type of protein that is naturally produced by the body. Levels of klotho, named after the mythological Greek figure Clotho, who spun the thread of human life, naturally decline as we get age. This process occurs at the same time we start experiencing age-related maladies like weaker bones, loss of muscle mass and declining cognitive abilities. Now, Spanish scientists have developed a klotho-based treatment that both increased levels of the protein in mice and boosted the lifespan of the rodents by a fifth. The experts said this is the equivalent of adding an extra 16 years to an 80 year-old human's lifespan. Not only did these mice live longer they showed improved muscle strength, bone density and brain function. While further research is needed before the treatment can be trialled on humans, the authors of the new study claim it could make a massive contribution to improving the ageing process. In the study, published in the journal Molecular Therapy, experts from the University of Barcelona used a specially modified virus to deliver klotho into the mice's cells. This harmless virus carried the biological blueprints for cells to produce the protein, enabling the mice to boost their klotho levels over a long period. Mice were given the virus via two injections—one into a vein in the body and the other directly into the brain. This enabled it to bypass the natural barrier that normally protects the vital organ and allowed scientists to examine klotho's effect on the brain. In total, three groups of mice were tested. One received the treatment at six months of age, another got the dose at 12 months, and a final group of six month old mice got a placebo to act as a control. Male mice injected at 12 months were found to live the longest (31.5 months on average), a fifth longer than the control group who only lasted 26.3 months. The average mouse lives for roughly 12 to 18 months. The researchers noted that klotho levels were the highest among the animals injected at one year old. Researchers also tested the mice on their physical fitness and found those in the treatment group performed better in experiments measuring coordination and muscle strength. Tissue analysis showed mice on klotho had less internal scarring on their muscles and were generally more muscular. Female mice on the treatment, however, did not see the same lifespan extension due to severe health complications that the scientists said were not related to the treatment. However they did develop stronger bones. Analysis of brain tissue also showed mice of both sexes showed signs they were developing new neurons in the hippocampus, an area of the brain closely linked to learning. This suggests the treatment could combat age-related dementia. Currently the implications for using the treatment in people remain limited. Mice aren't humans, and many drugs and treatments that have shown promise on rodents have failed to replicate this success when trialled on people. Also of note is that many of the tissue samples used in the analysis only came from a limited number of rodents, between three and four, which could limit the results. However, Joan Roig-Soriano, an expert in neuroscience and author of the new study, said techniques that can provide klotho to humans are already available. 'We now have viral vectors that can reach the brain after being administered intravenously, which would make it easier to safely transfer this therapy to humans,' he said. 'Another option would be to administer the protein directly as a drug instead of using viral vectors, but we still need to find an efficient way to deliver it and ensure it reaches the target organs,' he said. Previous research has shown klotho can improve brain function in old primates and 'de-age' the brains of mice by decades. Klotho was originally discovered by Japanese researchers who found that the amount produced by mice could affect how long the rodents lived.
