Evox Therapeutics provided GBP 655,000 in the form of Duchenne UK funding to support research into its exosimili-based therapeutic platform.
Duchenne is a very comprehensive, progressive, muscular-disrupting disorder caused by the lack of functional proteins of dystrophs for which there is currently no medication.
Delivery of dystrophine or its shorter variants in these patients has the potential to become a very effective treatment option for the condition, the company warns.
Evox is an engineering exosome, a natural vesicular body system that allows the diversified use of medicines reaching previously inaccessible tissues and compartments such as the passage of the brain barrier to the blood for the administration of drugs into the central nervous system, the intracellular delivery of proteins, and the additional – hepatic administration of RNA therapeutics .
The idea is to use modified exosomes to supply either a long lasting dolphin or its shorter version in the pre-clinical models of Duchenne.
"We will explore to evaluate the potential of our exothermic drug platform in order to provide a functional dystrophin that is missing or deficient in these patients," said Antonin de Fougerolles, Evox CEO.
"This work will also allow us to research targeted delivery of muscle exosomes that could benefit not only Duchenne patients, but also finally in patients with other musculoskeletal disorders."
"We are pleased to work with Evox to advance in this potentially exciting task to help in the field of muscle dystrophy of Duchenne," said Emily Crossley and Alex Johnson, co-presidents of Duchenne UK.
"One of the most demanding aspects of the use of viruses to provide gene therapy is that many patients can already have what is known as pre-existing antibodies – they are" resistant "to the virus – and therefore the replacement gene that the virus is carrying will never achieve your goal.
"Exosomes could provide a potentially new method for the effective, safe and multiple administration of genetic material that encodes muscle dystrophy without the problem of previous antibodies."