Avrion Therapeutics targets genetically-linked neurological disorders using precision gene therapy to enable long-term therapeutic solutions. Avrion has developed a first-in-class precision gene therapy platform to selectively target discrete cells in the brain including neurons and astrocytes. Our approach is designed to enhance efficacy of therapeutic gene delivery to specifically targeted nervous system cells and increase safety by limiting expression in other cell types.
Avrion Therapeutics targets genetically-linked neurological disorders using precision gene therapy to enable long-term therapeutic solutions. Avrion has developed a first-in-class precision gene therapy platform to selectively target discrete cells in the brain including neurons and astrocytes. Our approach is designed to enhance efficacy of therapeutic gene delivery to specifically targeted nervous system cells and increase safety by limiting expression in other cell types.
Avrion gene therapy technology utilises vectors to deliver therapeutic gene sequences to discrete cell types simultaneously in the central nervous system. In particular, while neurons are most obviously disrupted by neurodegenerative disease, we also target astrocytes in combination with neurons as a treatment approach for the fatal motor neuron disease ALS. Healthy astrocytes provide metabolic support to neurons and reduce excitotoxicity. Diseased astrocytes are not only diminished in this protective ability but can further exacerbate neurodegeneration by releasing factors promoting inflammation and cell death. By targeting expression of therapeutic gene sequences specifically to both neurons and astrocytes together from a single gene therapy vector, we observe significant synergistic therapeutic benefits in preclinical ALS models, when compared to seperate treatment of either cell type alone. Our nervous system cell-type specific vector technology has the added potential safety benefit of limiting unwanted vector expression outside of the desired targeted cells.
Avrion gene therapy technology utilises vectors to deliver therapeutic gene sequences to discrete cell types simultaneously in the central nervous system. In particular, while neurons are most obviously disrupted by neurodegenerative disease, we also target astrocytes in combination with neurons as a treatment approach for the fatal motor neuron disease ALS. Healthy astrocytes provide metabolic support to neurons and reduce excitotoxicity. Diseased astrocytes are not only diminished in this protective ability but can further exacerbate neurodegeneration by releasing factors promoting inflammation and cell death. By targeting expression of therapeutic gene sequences specifically to both neurons and astrocytes together from a single gene therapy vector, we observe significant synergistic therapeutic benefits in preclinical ALS models, when compared to seperate treatment of either cell type alone. Our nervous system cell-type specific vector technology has the added potential safety benefit of limiting unwanted vector expression outside of the desired targeted cells.
Avrion's first clinical candidate gene therapy product, AVR-001, is preparing to enter IND-enabling studies to treat patients with a form of the fatal motor neuron disease (amyotrophic lateral sclerosis - ALS or MND), linked to aberrations of the protein Superoxide Dismutase 1 - SOD1.
Avrion's first clinical candidate gene therapy product, AVR-001, is preparing to enter IND-enabling studies to treat patients with a form of the fatal motor neuron disease (amyotrophic lateral sclerosis - ALS or MND), linked to aberrations of the protein Superoxide Dismutase 1 - SOD1.
This therapeutic platform is supported by a preclinical discovery engine to identify novel targets to treat neurodegenerative diseases with unmet medical need. These targets will be addressed by our technology which allows cell-specific genetic manipulation within the brain and spinal cord.
This therapeutic platform is supported by a preclinical discovery engine to identify novel targets to treat neurodegenerative diseases with unmet medical need. These targets will be addressed by our technology which allows cell-specific genetic manipulation within the brain and spinal cord.
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