Patent No. 2023252508 Clears Path for Klotho Gene Therapy to Target ALS in Humans

For ALS patients and the clinicians treating them, therapeutic advances remain frustratingly slow. The latest milestone comes from an unlikely source—a mining company’s biotech division, which has secured a patent for a gene therapy that could address a fundamental aging-related mechanism, not just a specific mutation. While significant hurdles remain, the IP win validates a decade of research into the Klotho protein’s neuroprotective role.

Greenland Mines (Nasdaq: GRML), a company primarily known for mining, has taken a notable step into the biotechnology arena with the granting of Australian Patent No. 2023252508, covering its Klotho gene therapy technology, KLTO-202. This patent, invented by researchers at Universitat Autònoma de Barcelona and exclusively licensed by Greenland Mines’ Biotech Division, protects the use of a muscle-cell specific promoter to drive expression of the human Klotho s-KL protein for treating motor impairment. The claims are broad, encompassing neuronal cells, induced pluripotent stem cells (iPSCs), and multiple delivery systems including adeno-associated viral vectors (AAV) and non-viral vectors with muscle-cell or motor neuron-cell tropism. This IP reinforcement is critical for a preclinical program targeting amyotrophic lateral sclerosis (ALS) and other debilitating neuromuscular diseases.

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2023252508, covering its Klotho gene therapy technology, KLTO-202.

The Klotho protein has emerged as a molecule of interest in the anti-aging and neuroprotection fields. Preclinical studies have linked reduced Klotho levels to accelerated aging, cognitive decline, and motor neuron vulnerability. By delivering a gene that increases Klotho expression specifically in muscle cells or motor neurons, KLTO-202 aims to address a fundamental biological deficit rather than a single genetic mutation. This approach is differentiated from gene therapies like tofersen, which target a specific SOD1 mutation in a small subset of ALS patients. If the promising preclinical signals translate into human data, a Klotho-based therapy could offer a new treatment paradigm for a broader ALS population.

For Greenland Mines, the patent is a strategic asset that may help attract partners or investment to fund costly clinical development. The company’s core revenue comes from mining operations, and biotech ventures require substantial capital. CEO Dr. Joseph Sinkule emphasized that the company has advanced KLTO-202 over three years on a simple scientific premise, but the leap from patent to approved therapy is vast. Competitors in the ALS gene therapy space, such as Ionis/Biogen with tofersen and other programs targeting C9orf72 or ataxin-2, have far more resources and clinical data. Greenland’s broad patent claims could give it leverage if the mechanism proves biologically valid, but the field is littered with failed attempts.