Evox dystrophin exosome therapy

Evox Therapeutics developed an experimental exosome-based RNA therapy for the treatment of Duchenne muscular dystrophy (DMD). The platform utilizes engineered exosomes—naturally occurring extracellular vesicles—to deliver mRNA encoding dystrophin or its truncated functional variants, such as mini-dystrophin or micro-dystrophin, directly to muscle tissues. This approach was designed as a non-viral alternative to traditional gene therapy, potentially avoiding the immunogenicity issues associated with adeno-associated virus (AAV) vectors. Although the program demonstrated proof-of-concept in preclinical studies funded by the charity Duchenne UK, the collaboration ended in 2020, and the program is no longer featured in Evox's active development pipeline, suggesting it has been deprioritized or terminated.

Other names

dystrophin exosome-based therapyEvox DMD programDystrophin-Exosome-based therapy