Latest news with #neuroprotection
National Post
3 days ago
- Health
- National Post
Cognito Therapeutics to Present Five Abstracts at AAIC 2025 Highlighting Spectris™ Treatment in Alzheimer's Disease
Article content CAMBRIDGE, Mass. — Cognito Therapeutics, a clinical-stage medical device company pioneering non-invasive neuroprotection therapies against neurodegenerative diseases, today announced that five abstracts featuring its lead investigational device, Spectris™, have been accepted for presentation at the Alzheimer's Association International Conference (AAIC) 2025, taking place July 27–31 in Toronto, Canada. Article content The presentations will showcase clinical, neurophysiological, and biomarker data supporting Spectris™, an at-home device designed to deliver personalized, sensory-driven gamma stimulation to slow the progression of Alzheimer's disease. Article content Among the highlights is an oral presentation selected by the International Society to Advance Alzheimer's Research and Treatment (ISTAART): Article content Preservation of Neurophysiological Signals and White Matter with 40Hz Sensory Stimulation in Alzheimer's Disease Session: ISTAART Oral Session Presenter: Chandran Seshagiri, PhD Date/Time: Saturday, July 26, 2025, 10:15–11:15 AM Article content In addition, Cognito will present five poster sessions: Article content 'These presentations underscore the broad neuroprotective potential of Spectris, from preserving white matter and functional connectivity to measuring real-world benefits that matter to patients,' said Ralph Kern, MD, MHSc, Chief Medical Officer of Cognito Therapeutics. 'We look forward to sharing these latest insights with the global neuroscience community at AAIC.' Article content Spectris™ is currently being evaluated in the ongoing HOPE pivotal clinical trial (NCT05637801) for Alzheimer's disease. It is an investigational device and has not yet received regulatory approval for commercial use from the U.S. Food and Drug Administration or any other health authority. Article content About Cognito Therapeutics Article content Cognito Therapeutics is a late clinical-stage medical device company pioneering neuroprotective therapies to address the unmet needs of patients living with CNS disorders. Its lead product, Spectris™ AD, is an at-home therapeutic device that uses non-invasive, sensory-driven neurostimulation to evoke gamma frequency brain activity. The company's feasibility studies have shown the potential for Spectris AD to preserve cognition, daily function, and slow brain atrophy in patients diagnosed with mild-to-moderate AD. Cognito is headquartered in Cambridge, MA. For more information, visit and follow @cognitotx. Article content Article content Article content Article content Contacts Article content Media Contact Article content Article content Kimberly Ha Article content Article content Article content
Yahoo
30-06-2025
- Business
- Yahoo
Klotho Neurosciences Moves Forward with Manufacturing Gene Therapy for the Treatment of ALS
NEW YORK, June 30, 2025 /PRNewswire/ -- Klotho Neurosciences, Inc. (NASDAQ: KLTO) today announced that it is moving forward with manufacturing and process development work in preparation for clinical trials of KLTO-202, its investigational gene therapy for amyotrophic lateral sclerosis (ALS). A unique RNA splice variant of the human gene called alpha-Klotho has been licensed by the Company from the Autonomous University of Barcelona ("UAB") including patents, patent applications, research, knowhow and other intellectual properties for use in the development of advanced gene and gene-engineered cell therapies. The human alpha-Klotho gene is located in cells found throughout the human body. It is a five exon gene that produces two protein isoforms: a full-length protein found mainly in cells of the kidney called membrane-bound Klotho (or m-KL), which controls phosphate homeostasis, and a much smaller protein isoform called secreted alpha-Klotho (or s-KL). The s-KL RNA splice variant is found mainly in the brain and spinal cord neurons; the variant protein is neuroprotective by minimizing both oxidative stress and neuroinflammation. Animal studies supported by the Company over the past two years in mouse and non-human primate models of rapid aging, in models of human Alzheimer's disease, and in models of ALS have shown that over-expression and amplification of the tissue levels of s-KL using a gene therapy approach result in highly favorable therapeutic outcomes in every model tested. These results have been published in peer-reviewed scientific journals and now support the transition of KLTO-202 into the clinical development stage. The Company expects that it will take approximately eight months to complete process development and manufacturing of KLTO-202, and about four to six months to conduct meetings with FDA, complete all FDA-mandated animal safety studies, file an investigational new drug application (IND), train and prepare clinical sites where the Phase I/II studies can be conducted, and then begin the single-dose gene therapy studies in ALS patients by the third quarter of next year. The Company will work with contract research organizations (CROs) to facilitate all activities including manufacturing and clinical trials without the need to hire several dozen staff members, which would significantly increase our operating overhead. Dr. Joseph Sinkule, the Company's CEO and founder commented: "With our recent fundraising success, we're moving forward with manufacturing the s-KL transgene DNA for KLTO-202. We've identified a more efficient method of producing the AAV vector to deliver the s-KL gene directly to motor neurons—the cells most affected by ALS. Our goal is to increase local s-KL protein levels to protect these neurons from the damage that leads to voluntary and involuntary muscle paralysis and ultimately death." ALS typically progresses rapidly, with most patients losing mobility, respiratory function, and life within just 2–3 years of diagnosis. 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 its 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. Investor Contact and Corporate Communications - Jeffrey LeBlanc, CFOir@ 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. 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. SOURCE Klotho Neurosciences, Inc. 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
Associated Press
30-06-2025
- Business
- Associated Press
Klotho Neurosciences Moves Forward with Manufacturing Gene Therapy for the Treatment of ALS
NEW YORK, June 30, 2025 /PRNewswire/ -- Klotho Neurosciences, Inc. (NASDAQ: KLTO) today announced that it is moving forward with manufacturing and process development work in preparation for clinical trials of KLTO-202, its investigational gene therapy for amyotrophic lateral sclerosis (ALS). A unique RNA splice variant of the human gene called alpha-Klotho has been licensed by the Company from the Autonomous University of Barcelona ('UAB') including patents, patent applications, research, knowhow and other intellectual properties for use in the development of advanced gene and gene-engineered cell therapies. The human alpha-Klotho gene is located in cells found throughout the human body. It is a five exon gene that produces two protein isoforms: a full-length protein found mainly in cells of the kidney called membrane-bound Klotho (or m-KL), which controls phosphate homeostasis, and a much smaller protein isoform called secreted alpha-Klotho (or s-KL). The s-KL RNA splice variant is found mainly in the brain and spinal cord neurons; the variant protein is neuroprotective by minimizing both oxidative stress and neuroinflammation. Animal studies supported by the Company over the past two years in mouse and non-human primate models of rapid aging, in models of human Alzheimer's disease, and in models of ALS have shown that over-expression and amplification of the tissue levels of s-KL using a gene therapy approach result in highly favorable therapeutic outcomes in every model tested. These results have been published in peer-reviewed scientific journals and now support the transition of KLTO-202 into the clinical development stage. The Company expects that it will take approximately eight months to complete process development and manufacturing of KLTO-202, and about four to six months to conduct meetings with FDA, complete all FDA-mandated animal safety studies, file an investigational new drug application (IND), train and prepare clinical sites where the Phase I/II studies can be conducted, and then begin the single-dose gene therapy studies in ALS patients by the third quarter of next year. The Company will work with contract research organizations (CROs) to facilitate all activities including manufacturing and clinical trials without the need to hire several dozen staff members, which would significantly increase our operating overhead. Dr. Joseph Sinkule, the Company's CEO and founder commented: 'With our recent fundraising success, we're moving forward with manufacturing the s-KL transgene DNA for KLTO-202. We've identified a more efficient method of producing the AAV vector to deliver the s-KL gene directly to motor neurons—the cells most affected by ALS. Our goal is to increase local s-KL protein levels to protect these neurons from the damage that leads to voluntary and involuntary muscle paralysis and ultimately death.' ALS typically progresses rapidly, with most patients losing mobility, respiratory function, and life within just 2–3 years of diagnosis. 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 its 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. Investor Contact and Corporate Communications - Jeffrey LeBlanc, CFO [email protected] 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. 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.
Yahoo
30-06-2025
- Business
- Yahoo
Klotho Neurosciences Moves Forward with Manufacturing Gene Therapy for the Treatment of ALS
NEW YORK, June 30, 2025 /CNW/ -- Klotho Neurosciences, Inc. (NASDAQ: KLTO) today announced that it is moving forward with manufacturing and process development work in preparation for clinical trials of KLTO-202, its investigational gene therapy for amyotrophic lateral sclerosis (ALS). A unique RNA splice variant of the human gene called alpha-Klotho has been licensed by the Company from the Autonomous University of Barcelona ("UAB") including patents, patent applications, research, knowhow and other intellectual properties for use in the development of advanced gene and gene-engineered cell therapies. The human alpha-Klotho gene is located in cells found throughout the human body. It is a five exon gene that produces two protein isoforms: a full-length protein found mainly in cells of the kidney called membrane-bound Klotho (or m-KL), which controls phosphate homeostasis, and a much smaller protein isoform called secreted alpha-Klotho (or s-KL). The s-KL RNA splice variant is found mainly in the brain and spinal cord neurons; the variant protein is neuroprotective by minimizing both oxidative stress and neuroinflammation. Animal studies supported by the Company over the past two years in mouse and non-human primate models of rapid aging, in models of human Alzheimer's disease, and in models of ALS have shown that over-expression and amplification of the tissue levels of s-KL using a gene therapy approach result in highly favorable therapeutic outcomes in every model tested. These results have been published in peer-reviewed scientific journals and now support the transition of KLTO-202 into the clinical development stage. The Company expects that it will take approximately eight months to complete process development and manufacturing of KLTO-202, and about four to six months to conduct meetings with FDA, complete all FDA-mandated animal safety studies, file an investigational new drug application (IND), train and prepare clinical sites where the Phase I/II studies can be conducted, and then begin the single-dose gene therapy studies in ALS patients by the third quarter of next year. The Company will work with contract research organizations (CROs) to facilitate all activities including manufacturing and clinical trials without the need to hire several dozen staff members, which would significantly increase our operating overhead. Dr. Joseph Sinkule, the Company's CEO and founder commented: "With our recent fundraising success, we're moving forward with manufacturing the s-KL transgene DNA for KLTO-202. We've identified a more efficient method of producing the AAV vector to deliver the s-KL gene directly to motor neurons—the cells most affected by ALS. Our goal is to increase local s-KL protein levels to protect these neurons from the damage that leads to voluntary and involuntary muscle paralysis and ultimately death." ALS typically progresses rapidly, with most patients losing mobility, respiratory function, and life within just 2–3 years of diagnosis. 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 its 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. Investor Contact and Corporate Communications - Jeffrey LeBlanc, CFOir@ 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. 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. View original content to download multimedia: Sign in to access your portfolio
The Australian
20-06-2025
- Health
- The Australian
Neurizon's NUZ-001 shows promise in Huntington's disease
Neurizon's NUZ-001 and active metabolite NUZ-001 Sulfone show strong neuroprotective effects in a zebrafish Huntington's model Results show potential of drug to counteract early neurodegenerative damage caused by disease Neurizon plans to initiate additional validation studies in mammalian models of Huntington's Special Report: Clinical-stage biotech Neurizon Therapeutics has reached a milestone in development of its lead drug candidate NUZ-001 to treat Huntington's disease. Neurizon Therapeutics (ASX:NUZ), which is dedicated to advancing treatments for neurodegenerative diseases, has rolled out new preclinical data demonstrating significant neuroprotective effects of NUZ-001 and its active metabolite NUZ-001 Sulfone, in a zebrafish model of Huntington's disease. Huntington's disease is a rare, inherited neurodegenerative disorder that causes progressive degeneration of motor function, cognition and mental health. The disease affects between 2.7 and 4.8 per 100,000 people globally with no cure and no disease-modifying treatment. The treatments available only manage symptoms. In the Huntington's disease model, targeted mRNA knockdown of the Htt (huntingtin) protein triggered hallmark disease characteristics, including: Increased cell death Morphological malformations (smaller eyes and swollen hindbrains) Impaired haemoglobin production and Reduced expression of brain-derived neurotrophic factor (BDNF), a critical biomarker of neuronal function and survival. mRNA knockdown is a lab technique that reduces the activity of a specific gene, in this case the HTT gene, which produces the protein involved in Huntington's disease. Treatment with either NUZ-001 or NUZ-001 Sulfone after Htt knockdown: Prevented developmental and morphological abnormalities Attenuated neuronal cell death Restored the delayed production of haemoglobin; and Rescued BDNF expression. Neurizon said the results provided evidence of NUZ-001 and NUZ-001 Sulfone's potential to counteract early neurodegenerative damage. Study details For the preclinical study, wild-type zebrafish embryos were raised in standard conditions. Morpholino antisense oligonucleotides (MOs) targeting Htt mRNA were then injected into one-cell stage embryos to decrease Htt expression. NUZ-001 or NUZ-001 Sulfone at 1 and 10 μM concentrations were added to the embryonic media six hours post-fertilisation to evaluate the protective effects on Htt knockdown-induced deficits. At two days post-fertilisation changes in morphology (eye size and hindbrain swelling), neuronal cell death (apoptosis), haemoglobin levels, and the BDNF expression levels were analysed. Source: Neurizon Therapeutics Knockdown of Htt (Htt MO) resulted in smaller eyes and swollen hindbrain ventricles in zebrafish embryos. Neurizon said partial rescue of eye size and full reversal of hindbrain swelling were observed with 10 μM NUZ-001 and NUZ-001 Sulfone (Figure 2b). Source: Neurizon Therapeutics Other key findings of the study include: Neuronal cell death was significantly higher in the Htt knockdown group, while treatment with 1 μM and 10 μM NUZ-001, and 10 μM NUZ-001 Sulfone, significantly reduced apoptosis Haemoglobin levels were significantly decreased in the Htt knockdown group but partially restored by both concentrations of NUZ-001 and NUZ-001 Sulfone; and Expression of BDNF transcripts was significantly rescued with 10 μM NUZ-001 and 10 μM NUZ-001 Sulfone. Watch: Last patient completes treatment in OLE study NUZ-001 showing promise NUZ-001 is currently in clinical development for the most common form of motor neurone disease (MND) called amyotrophic lateral sclerosis (ALS), where it has shown: Preclinical efficacy in enhancing proteostasis Reducing pathological protein aggregation; and Preserving neuronal function. The company said new findings in the Huntington model further underscore NUZ-001's potential as a platform therapy targeting core cellular stress and clearance mechanisms common to multiple neurodegenerative diseases. Neurizon plans to advance additional preclinical studies in mammalian models of Huntington's disease as part of its broader strategy to expand NUZ-001's therapeutic applications to other progressive neurological disorders with high unmet need. 'These results mark another important milestone in the realisation of the potential for NUZ-001 to treat a range of neurodegenerative diseases,' CEO and managing director Dr Michael Thurn said. 'Huntington's disease is a devastating, rare genetic disorder that causes the progressive breakdown of nerve cells in the brain, leading to a range of symptoms including uncontrolled movements, cognitive decline, and emotional disturbances. 'These exciting results demonstrate NUZ-001 has consistent neuroprotective effects beyond amyotrophic lateral sclerosis (ALS), strengthening our conviction in NUZ-001's potential as a disease-modifying platform therapy across a range of neurodegenerative conditions.' This article was developed in collaboration with Neurizon Therapeutics, a Stockhead advertiser at the time of publishing. This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.
