Klotho Neurosciences ALS gene therapy KLTO-202 is advancing through a critical phase of manufacturing and process development, positioning the company for early-stage clinical trials by Q3 2026. This significant milestone reflects Klotho Neurosciences, Inc. (NASDAQ: KLTO)’s ongoing commitment to tackling amyotrophic lateral sclerosis (ALS), a debilitating neurodegenerative condition.
Proprietary Gene Licensed for Advanced Therapies
The company recently secured exclusive rights from the Autonomous University of Barcelona for a unique RNA splice variant of the human alpha-Klotho gene. This agreement covers essential patents and scientific know-how, enabling Klotho Neurosciences to engineer novel gene and cell therapies. The alpha-Klotho gene produces two isoforms: membrane-bound Klotho, which regulates phosphate levels in the kidneys, and the secreted alpha-Klotho (s-KL) variant, which predominates in brain and spinal neurons. The s-KL protein is known for its neuroprotective role, mitigating oxidative stress and neuroinflammation, two key contributors to ALS progression.
Preclinical Evidence Supports Clinical Transition
Encouraging data from animal models underpins the company’s strategy. Over the past two years, research involving mice and non-human primates has demonstrated that enhancing s-KL expression through gene therapy yields positive outcomes in models of rapid aging, Alzheimer’s disease, and ALS. These findings, published in reputable journals, validate the potential of KLTO-202 as a disease-modifying treatment.
Klotho Neurosciences ALS Gene Therapy Timeline and Strategic Execution
Klotho Neurosciences ALS gene therapy programme anticipates eight months for manufacturing and process refinement, followed by four to six months dedicated to regulatory engagement and investigational new drug (IND) filing. The company intends to collaborate with contract research organisations (CROs) for manufacturing and trial management, maintaining operational agility without significantly increasing internal headcount.
Dr. Joseph Sinkule, CEO and founder, highlighted the firm’s operational discipline: “Following our recent capital raise, we are focused on producing the s-KL transgene DNA for KLTO-202 and refining our AAV vector delivery method. Our goal is to elevate s-KL protein levels within motor neurons, protecting them from ALS-induced degeneration.”
Addressing a Critical Unmet Need
ALS remains an aggressive disease with limited therapeutic options, often leading to severe paralysis and mortality within a few years of diagnosis. By directly targeting the neurodegenerative mechanisms through gene therapy, Klotho Neurosciences aims to extend patient mobility and quality of life.
