Evox Therapeutics has received £ 655,000 of Duchenne UK funding to support the search for an exosomal-based therapeutic platform for the disease.
Duchenne is a highly debilitating, progressive, musculoskeletal disorder due to a lack of functional dystrophin protein, which is currently not a cure.
Delivering distrophine or its short formulations in these patients can be a very effective treatment option for diagnosing the disease.
Evox is an engineer exosome, a natural hydric vesicle delivery system for a variety of drugs to access previously unavailable tissues and compartments such as blood brain barriers, drug delivery to the central nervous system, intracellular delivery of proteins, and additional medications for RNA therapeutic agents.
The idea is to use custom exosomes to deliver either full length dystrophin or its shorter variants to Duchenne preclinical models.
"We undertake research to evaluate the potential of the exosome drug base to deliver functional dystrophin lacking or lacking in these patients," said Antonin de Fougerolles, Managing Director of Evox.
"With this work, we can also investigate the targeted distribution of exosomes intramuscularly, which can be useful not only for Duchenne patients, but ultimately for patients with other musculoskeletal disorders."
"We are delighted to work with Evox to promote this potentially exciting work in the Duchenne Muscle Dystrophy," says Emily Crossley and Alex Johnson, Secretary General of Duchenne UK.
"One of the most challenging aspects of viral use to produce gene therapy is that many patients already have known as existing antibodies – they are" resistant "to the virus – and the virus-transported gene will never achieve its goal.
"Exosomes could provide a potential new method for coding dystrophin in genetic material efficiently, safely and repeatedly in muscle without the problem of existing antibodies."