Evox Therapeutics has secured £655,000 from Duchenne UK to explore its exosome-based therapeutic platform to deliver either full-length dystrophin or its shorter variants in pre-clinical models of Duchenne muscular dystrophy (Duchenne).
Duchenne is a highly debilitating, progressive, muscle-wasting disorder caused by the lack of functional dystrophin protein, for which there is currently no cure. Delivery of dystrophin or its shorter variants to these patients has the potential to be a highly effective treatment option in Duchenne.
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, intracellular delivery of proteins, and extra-hepatic delivery of RNA therapeutics.
Evox is developing its own proprietary pipeline of exosome-based therapeutics for the treatment of rare, life-threatening diseases with significant unmet need.
CEO Antonin de Fougerolles said: “We will conduct research to assess the potential of our exosome drug platform to deliver functional dystrophin which is missing or defective in these patients.
“This work will also allow us to explore targeted delivery of exosomes to muscle which may be beneficial not only for Duchenne patients, but also ultimately for patients with other musculoskeletal diseases.”
Emily Crossley & Alex Johnson, Co-CEOs of Duchenne UK, added: “One of the most challenging aspects of using viruses to deliver gene therapy is that many patients may already have what are known as pre-existing antibodies –they are ‘resistant’ to the virus – and so the replacement gene carried by the virus will never reach its target.
“Exosomes could provide a potential new method for effectively, safely, and repeatedly delivering genetic material encoding for dystrophin to muscles without the problem of pre-existing antibodies.”