Avrion Therapeutics is a spin-off from the Swiss Federal Institute of Technology in Lausanne (EPFL) developing innovative precision gene therapies for neurodegenerative disorders. Avrion’s approach is based on cell-type specific vector-based solutions to target discrete Central Nervous System (CNS) cell populations. This results in synergistic neuroprotection, while simultaneously enhancing safety.  Avrion’s first gene therapy candidates target Amyotrophic Lateral Sclerosis (ALS), a fatal motor neuron disease (MND).
Investigation of ALS pathogenic mechanisms has mostly been focused on motor neurons so far. However, emerging evidence supports that non-neuronal cells (i. e. astrocytes, microglia) play a critical role in the disease, through reshaping neuronal synaptic connections. To address this pathology, Avrion has engineered two candidate gene therapy products referred to as AVR-001 and AVR-002. 
The first candidate (AVR-001) harnessed an advanced vector platform designed to selectively and simultaneously target ALS associated with mutations (or pathogenic misfolding) of SOD1 in both neurons and astrocytes. AVR-001 has been validated as potently effective and safe in both rodent and non-human primate models.  
Our second candidate gene therapy product (AVR-002) addresses all forms of ALS. AVR-002 is designed to engage a novel neuroimmune pathophysiological mechanism which occurs in both familial and sporadic forms of ALS, plus potentially other neurodegenerative disorders (like frontotemporal dementia). This wholly novel disease mechanism emerged from a suppressor screen using our discovery platform, which exploits humanized Drosophila as disease models. The uniqueness of AVR-002 therapeutic solution is to combine (i) vector-based targeting of glial cells, and (ii) protein engineering to deliver a cell-type selective neuro-immunomodulatory factor that can protect neuronal synaptic connections in ALS.

Avrion Therapeutics

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