Evox Therapeutics has secured £ 655,000 in funding from Duchenne UK, to support exploration of its ex-based therapeutic platform for the state.
Duchenne is a very debilitating, progressive, muscle-spreading 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 very effective treatment option for the condition noted by the company.
Evox is an engineer sexosome, the body's natural vesicular delivery system, to enable a wide range of drugs to reach previously unavailable tissues and compartments, such as transfer of the blood brain barrier to deliver central nervous system, intracellular delivery of proteins and extrahepatic delivery of RNA therapeutic agents.
The idea is to use manipulated exosomes to deliver either full-length dystrophins or its shorter variants in preclinical models of Duchenne.
"We will do research to assess the potential of our exotic drug platform to deliver functional dystrophy that is missing or defective in these patients," said Antonin de Fougerolles, Evox's CEO.
"This work will also enable us to explore targeted deliveries of exosomes to muscles that may be useful not only to Duchenne patients, but also to patients with other musculoskeletal diseases."
"We are excited to work with Evox to promote this potentially exciting work to assist you in Duchenne Muscular Dystrophy," added Emily Crossley and Alex Johnson, CEO of Duchenne UK.
"One of the most challenging aspects of using viruses to deliver gene therapy is that many patients already have what is known as existing antibodies – they are resistant to the virus – and so, the virus carried by the virus will never reach its target.
"Exosomes can provide a potential new method for efficiently, safely and repeatedly delivering genetic material encoding dystrophin into muscles without problems with existing antibodies."