science

Avrion Therapeutics targets 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. Our approach is designed to enhance efficacy of therapeutic gene delivery to the nervous system and increase safety by limiting expression in other tissues. Furnished by targets from our discovery engine, we have deployed this platform initially to develop new gene therapies to treat ALS, a fatal neurodegenerative disease.

TECHNOLOGY PLATFORM

Avrion gene therapy platform technology utilises vectors to deliver therapeutic gene sequences to discrete cell types simultaneously in the central nervous system (CNS). In particular, while neurons are most obviously disrupted by neurodegenerative disease, we also target astrocytes and other CNS cells in combination with neurons as a treatment approach for the fatal motor neuron disease ALS. 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 SOD1 ALS models, when compared to separate treatment of either cell type alone. Our cell-type specific expression technology can also be used to produce secreted therapeutic factors in the CNS to engage other disease relevant cell types. 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.

PRODUCT DEVELOPMENT

AVR-001: 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.

AVR-002: Avrion's second candidate gene therapy product, AVR-002, which engages a novel neuroimmune disease mechanism, is designed to treat all ALS patients with either familial or sporadic disease.

DISCOVERY ENGINE

Our therapeutic platform is supported by a preclinical discovery engine in partnership with EPFL designed to identify novel targets to treat neurodegenerative diseases with unmet medical need. Human disease genetics provides a foundation to generate, using genome engineering technology, humanised Drosophila in vivo models. Exploiting the high-throughput genetics of this system together with machine vision and AI-assisted phenotypic screening enables the identification of disease modifying genetic and cellular targets. These targets can then be addressed by our technology which allows cell-specific genetic manipulation within the brain and spinal cord.

TECHNOLOGY PLATFORM

Avrion gene therapy platform technology utilises vectors to deliver therapeutic gene sequences to discrete cell types simultaneously in the central nervous system (CNS). In particular, while neurons are most obviously disrupted by neurodegenerative disease, we also target astrocytes and other CNS cells in combination with neurons as a treatment approach for the fatal motor neuron disease ALS. 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 SOD1 ALS models, when compared to separate treatment of either cell type alone. Our cell-type specific expression technology can also be used to produce secreted therapeutic factors in the CNS to engage other disease relevant cell types. 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.

PRODUCT DEVELOPMENT

AVR-001: 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.

AVR-002: Avrion's second candidate gene therapy product, AVR-002, which engages a novel neuroimmune disease mechanism, is designed to treat all ALS patients with either familial or sporadic disease.

DISCOVERY ENGINE

Our therapeutic platform is supported by a preclinical discovery engine in partnership with EPFL designed to identify novel targets to treat neurodegenerative diseases with unmet medical need. Human disease genetics provides a foundation to generate, using genome engineering technology, humanised Drosophila in vivo models. Exploiting the high-throughput genetics of this system together with machine vision and AI-assisted phenotypic screening enables the identification of disease modifying genetic and cellular targets. These targets can then be addressed by our technology which allows cell-specific genetic manipulation within the brain and spinal cord.

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