Novel Swiss gene therapy to treat neurodegenerative disease ALS
Avrion Therapeutics, a young EPFL spin-off, has developed an innovative gene therapy to tackle certain genetically defined forms of the deadly ALS disease.
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Amyotrophic lateral sclerosis (ALS) or motor neuron disease (MND) is a deadly neurodegenerative disease that leads to complete paralysis and death of patients within 2 to 5 years. According to the ALS Association, someone is diagnosed with ALS every 90 minutes and around 450,000 people worldwide are suffering from the condition. To raise awareness of this disease, which kills yearly around 100,000 people a year, the viral Ice Bucket Challenge was launched in 2014 to encourage donations to research.
Innovative gene therapy for brain cells
Since 2020, the Basel-based start-up Avrion Therapeutics has been bringing hope to patients. Avrion has developed a first-in-class precision gene therapy platform based on novel vectors to selectively and safely target discrete cells in the brain, including neurons and astrocytes. The idea for this novel therapy is based on a decade of research at EPFL, in the Brain Mind Institute and the Campus Biotech Gene Therapy Platform (EPFL).
Improving the lifespan of ALS patients
The Avrion team, together with scientists working with Professor Brian McCabe from the Brain Mind Institute at EPFL, have developed technologies that allows to replace genes in a small animal model, the fruit fly Drosophila, with human genes including defective human genes associated with ALS. According to McCabe, the results so far are promising. The research group has identified two novel genetic targets that, when manipulated, significantly improve the lifespan of an ALS fruit fly model. They are confident that better understanding these targeted genes can produce a breakthrough in understanding the disease and enable new treatments using gene therapy.
Gene therapy offers the opportunity to rapidly move from discoveries made in animal models to treatments that could be evaluated in patients. The technology has the potential advantages of being faster to develop and may produce fewer side effects than conventional drugs. A single gene therapy treatment normally brings long-term benefits
Preparing for clinical first-in-human trials
Avrion's first clinical candidate gene therapy product, AVR-001, is preparing to enter the IND programme to secure approval to conduct the first-in-human clinical trials and therefore to treat patients with a fatal neuromuscular disorder. The technology developed by Avrion can also be deployed to treat other neurodegenerative disorders, giving hope to many more patients worldwide.
The spin-off has received support from the Swiss Innovation Agency Innosuisse and is also part of Venture Leaders Biotech 2021, a Swiss national start-up team specialised in the biotech sector.