Avrion Therapeutics targets genetically-linked neurodegenerative diseases using precision gene therapy to enable long-term solutions. Avrion has developed a first-in-class precision gene therapy platform to selectively and safely target discrete cells in the brain including neurons and astrocytes.
Avrion gene therapy technology utilises vectors to deliver therapeutic gene sequences to discrete cell types in the central nervous system. Gene therapy can provide neuroprotection through the replacement, addition, silencing or editing of genetic information. Gene therapy typically requires a single administration of the treatment.
Avrion's first clinical candidate gene therapy product, AVR-001, is preparing to enter IND-enabling studies to treat patients with a form of motor neuron disease (amyotrophic lateral sclerosis - ALS or MND), a fatal neuromuscular disorder.
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.