Klotho Neurosciences Secures Australian Patent for Novel Gene Therapy Targeting Motor Neuron Diseases
Key Insights
Klotho Neurosciences (search) received approval for 18 patent claims in Australia covering the use of human secreted Klotho protein (search) (s-KL) for treating motor impairment (search) and neuromuscular diseases.
The patent encompasses innovative delivery systems including muscle-cell specific promoters, induced pluripotent stem cells, and adeno-associated viral vectors for targeted therapeutic applications.
The technology addresses age-related decline in Klotho protein levels caused by DNA methylation, potentially offering new treatment options for conditions like ALS, muscular dystrophy (search), and spinal muscular atrophy (search).
Klotho Neurosciences (search), Inc. (Nasdaq: KLTO) announced that IP Australia (search) has accepted 18 patent claims for the composition and use of human secreted Klotho protein (search) (s-KL) isoform, marking a significant milestone in the company's development of gene therapies for motor neuron diseases. The patent, filed in collaboration with technology licensor Universitat Autonoma de Barcelona (search), covers innovative approaches to treating motor impairment (search) through targeted protein supplementation.
Patent Coverage and Technology Platform
The 18 approved claims encompass the use of a human muscle-cell specific promoter operatively linked to a nucleic acid gene sequence encoding the human s-KL protein (search) for treating motor impairment (search). The patent also covers advanced cellular delivery systems, including neuronal cells and induced pluripotent stem cells (iPSC) containing the muscle-cell specific promoter and s-KL nucleic acid gene sequence.
The technology platform extends to sophisticated delivery mechanisms, including adeno-associated viral vectors (AAV) and non-viral vectors with muscle-cell and motor neuron-cell tropism. These delivery systems are designed to target specific cell types affected by neuromuscular disorders, potentially improving therapeutic efficacy while minimizing off-target effects.
Addressing Age-Related Klotho Decline
Dr. Joseph Sinkule, founder and CEO of Klotho Neurosciences (search), explained the scientific rationale behind the approach: "As people age, they naturally lose innate, in vivo therapeutic concentrations of Klotho due to the commonly occurring DNA methylation of the promoter and gene leading to the 'silencing' of the Klotho gene (search) in neuromuscular ('motor neuron') cells and tissues in our body."
This age-related decline in Klotho protein levels represents a potential therapeutic target for addressing motor impairment (search). The company's approach focuses on therapeutic replacement or supplementation of human Klotho levels to counteract this natural decline and potentially restore motor function.
Broad Therapeutic Applications
The patent's "method of use" component covers treatment for subjects with diseases or disorders characterized by motor impairment (search), encompassing a wide range of neuromuscular and motor neuron diseases. The covered conditions include amyotrophic lateral sclerosis (search) (ALS), Charcot-Marie-Tooth disease, multiple sclerosis (search), muscular dystrophy (search) (both Duchenne and Becker muscular dystrophy), myasthenia gravis (search), myopathy, myositis, peripheral neuropathy, and spinal muscular atrophy (search) (SMA).
Additional applications extend to traumatic nerve injury, diabetic neuropathy, motor skills disorder, spinal cord injury, peripheral nerve injury, hereditary myopathy, toxic neuropathy, autoimmune peripheral polyneuropathy, acute inflammatory demyelinating polyradiculoneuropathy (AIDP), chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), drug-induced myopathy, sarcopenia, cachexia, age-related muscular atrophy, type II muscle fiber atrophy, and acquired autoimmune primary muscle disorders.
Specific motor neuron disorders covered include ALS, progressive bulbar palsy (PBP), pseudobulbar palsy, progressive muscular atrophy (PMA), primary lateral sclerosis (PLS), SMA, and monomelic amyotrophy (MMA).
Strategic Implications
Sinkule emphasized the strategic importance of the patent approval: "These claims are an important expansion of our patent estate, which we believe will help to provide momentum and security to pursue the therapeutic replacement or supplementation of human Klotho levels." The CEO described the company's approach to utilizing s-KL protein (search) as "a significant innovation" in addressing Klotho supplementation.
The patent strengthens Klotho Neurosciences (search)' position as it develops innovative, disease-modifying cell and gene therapies using proteins derived from the "anti-aging" human Klotho gene (search). The company's broader pipeline focuses on transforming treatment approaches for neurodegenerative disorders including ALS, Alzheimer's disease (search), Parkinson's disease (search), and age-related neuronal, ocular, skeletal, and muscular pathologies.
Technology Development Platform
Klotho Neurosciences (search) operates as a gene and cell therapy company utilizing tissue-specific promoters and novel tissue and organ delivery systems alongside the s-KL protein (search) technology. This integrated approach aims to improve the precision and efficacy of therapeutic interventions for neurodegenerative conditions.
The Australian patent approval represents a key milestone in the company's intellectual property strategy, providing protection for its core technology platform in a significant international market. The patent coverage encompasses both the composition of matter and methods of use, offering comprehensive protection for the company's therapeutic approach.