Avrion^TX

Avrion Therapeutics

Correct for Life

Mission

Pioneering synapse therapies for neurodegenerative disease

Avrion Therapeutics was founded in 2020 based on more than 10 years of research at the Brain Mind Institute at EPFL, the Swiss Federal Institute of Technology Lausanne, led by the groups of Dr. Bernard Schneider and Prof. Brian McCabe. Central to this research was the discovery that protecting synapses, the connections that enable communication among neurons in the brain and between the brain and the body, is essential to combating neurodegenerative disease.

Avrion is focused on preserving motor synapses in Amyotrophic lateral sclerosis (ALS). Also known as motor neuron disease (MND), Lou Gehrig’s disease or maladie de Charcot, ALS is a progressive and ultimately fatal neurodegenerative disorder of the motor system affecting approximately 300,000 people worldwide. Synapse loss is an early and critical event in the progressive deterioration of motor function across all forms of ALS. Preserving vulnerable synapses offers a new strategy for the treatment of ALS.

Avrion is also investigating synapse protection as a treatment for frontotemporal dementia (FTD), a group of progressive neurodegenerative disorders affecting behaviour, personality, language, and executive function. FTD is closely related to ALS through shared genetic and pathological features. It is a major cause of early-onset dementia, affecting approximately 700,000 people worldwide.

Pipeline

AVR-001: Avrion's first development candidate, AVR-001, is preparing to enter IND-enabling studies to treat patients with a form of ALS linked to aberrations of the protein Superoxide Dismutase 1 - SOD1.

AVR-002: Avrion's second development candidate, AVR-002, engages a novel neuroimmune disease mechanism that induces synapse loss in ALS and FTD. AVR-002 is designed to treat all ALS patients of either familial or sporadic disease origin.

AVR-003: Avrion's third development candidate, AVR-003, is designed to inhibit a neuronal factor which marks synapses for aberrant removal in ALS and FTD. Advancement of AVR-003 is ongoing.

Technology

Discovery Engine: Avrion’s therapeutic platform is supported by a preclinical discovery engine designed to identify novel targets for neurodegenerative diseases with significant unmet medical need. Guided by human disease genetics, genome-engineering technologies are used to generate humanised in vivo models [read more here] Combining the high-throughput capabilities of this system with machine vision and AI-assisted screening technologies enables the identification of disease-modifying genetic and cellular targets.

Targeted Delivery Platform: Avrion’s targeted delivery technology uses vectors to deliver therapeutic gene sequences simultaneously to specific cell types within the central nervous system (CNS). Although neurons are prominently affected in neurodegenerative diseases, astrocytes and other CNS cell types can also be impacted alongside neurons. Using a single product to direct therapeutic gene expression specifically to both neurons and astrocytes produces an enhanced, synergistic therapeutic benefits. Our cell-type-specific expression technology can also produce secreted therapeutic factors within the CNS, engaging additional disease-relevant cell types while offering the added safety benefits of limiting unwanted expression outside the CNS.

We are grateful for the support of these partners

Enquiries

contact@avriontx.com

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