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Evox Therapeutics Ltd (‘Evox’ or the ‘Company’), a leading exosome therapeutics company, today announces that it has secured £1.5 million in funding from Innovate UK, the UK’s Innovation Agency, through the Biomedical Catalyst Early Stage Award. The funds will be used to support the Company’s pre-clinical development of an exosome-based therapy to treat argininosuccinic aciduria (ASA), a rare life-threatening metabolic disease, in collaboration with University College London (UCL).

ASA is a rare genetic disorder characterised by deficiency or lack of the enzyme argininosuccinate lyase (ASL). ASL is central to two metabolic pathways: i) the liver-based urea cycle, which detoxifies ammonia, and ii) the citrulline-nitric oxide cycle, which synthesises nitric oxide from L-arginine. Patients with ASL deficiency can present either shortly after birth or later in life and are characterised by hyperammonaemia and a multi-organ disease with a severe neurological phenotype. ASA is the second most common urea cycle disorder and with merely symptomatic treatment available today; there is currently a significant unmet medical need for these patients.

Evox is engineering exosomes, the body’s natural vesicular delivery system, to enable a wide variety of drugs to reach previously inaccessible tissues and compartments, such as crossing the blood-brain barrier to deliver drugs to the central nervous system, enabling intracellular delivery of biologics, and allowing for extra-hepatic delivery of RNA therapeutics. UCL has world-leading expertise in urea cycle disorders and will perform testing of Evox’s exosome-based therapy in an in vivo ASL deficiency model.

Dr Per Lundin, Chief Operating Officer and Co-Founder of Evox, commented: “We are delighted that Innovate UK have chosen to support our work to develop an exosome-based therapy to treat patients with ASA. We intend to leverage the broad tissue distribution pattern of exosomes to deliver the fully functional enzyme to the liver and other key tissues, such as the central nervous system, in order to treat all aspects of the disease. We look forward to working with our academic collaborators at UCL who are at the forefront of developing pre-clinical models of the disease and treating patients.”

Dr. Paul Gissen from the UCL Great Ormond Street Institute of Child Health said: “We are very excited to receive this collaborative grant with Evox from Innovate UK. It will allow us to test the potential effect of this novel exosome-based therapy in a model of severe metabolic disease, argininosuccinic aciduria. Patients with this disorder suffer from dysfunction of the liver, brain and other organs and are representative of a larger group of inherited diseases in which there is a huge unmet need for disease-specific treatments. Therefore, if successful, the same approach may be used to treat many other severe illnesses.”


SOURCE: Evox Therapeutics