Klotho Neurosciences is advancing its gene therapy, KLTO-202, for the treatment of amyotrophic lateral sclerosis (ALS). The company is moving forward with manufacturing and process development, targeting clinical trials within the next year.

KLTO-202 utilizes a unique RNA variant of the human Klotho gene, shown in preclinical studies to be neuroprotective by reducing oxidative stress and inflammation. Positive results were observed in models of ALS, Alzheimer’s, and rapid aging.

Klotho anticipates completing manufacturing in eight months, followed by FDA meetings, safety studies, and clinical site preparation. Phase I/II trials are projected to begin in the third quarter of next year. The company will leverage contract research organizations to streamline development without significant staffing increases.

The therapy aims to increase levels of the s-KL protein in motor neurons, protecting them from the damage that leads to muscle paralysis in ALS patients. ALS is a rapidly progressing disease, with a typical life expectancy of 2-3 years post-diagnosis. Klotho Neurosciences is focused on developing disease-modifying therapies for neurodegenerative disorders.