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Klotho Neurosciences Moves Forward with Manufacturing Gene Therapy for the Treatment of ALS
  • Mon, 30 Jun 2025    5 Min Read
Klotho Neurosciences Moves Forward with Manufacturing Gene Therapy for the Treatment of ALS

NEW YORK, June 30, 2025 -- 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.