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Telomerase gene therapy for Alzheimer's disease

Telocyte is a company founded by Michael Fossel, MD, Ph.D., and Peter Rayson and is focused on curing Alzheimer’s disease using telomerase gene therapy.

Telomere erosion is one of the Hallmarks of Aging, and some scientists believe it is one of the major reasons we age, as we discuss here. Telocyte proposes to use telomerase gene therapy to restore critically short telomeres in target cells in order to potentially reverse Alzheimer’s pathology. In addition to the better-known effects of telomere erosion (cellular senescence and loss of tissue regeneration), the company believes that telomere erosion in glial cells slows the rate of molecular turnover that results from cell division, leading to an accumulation of molecular damage and increasingly dysfunctional cells that are unable to take up and degrade the hallmark molecular pathology of the disease: beta-amyloid, aberrant tau, and other extra- and intracellular aggregates. Thus, they hypothesize that telomerase gene therapy may restore glial function and physiologically clear molecular damage in the brain, thus preventing and reversing the disease.

Telocyte has signed a Memorandum of Understanding with the CNIO (Centro Nacional de Investigaciones Oncológicas) in Spain for collaboration to take telomerase gene therapy.to FDA human trials. Under the supervision of Dr. Maria Blasco, the CNIO has recently made advances in both animal research and delivery systems and is collaborating with Telocyte to bring telomerase therapy closer to the clinic.

The Telocyte company has stated that it has substantial funding to cover the costs of human trials and is awaiting an FDA decision on their IND application. According to their Q1 2021 newsletter, they had intended to move into large animal models by the end of that year; however, it does not appear that this has happened, and the most recent corporate newsletter is from Q4 of 2024, with no suggestion that such studies are underway or imminent. We judge it possible that the company is inactive.

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