Non-viral delivery for gene therapy

The Duchenne Research Fund committed £320,000 to accelerate research into alternative ways to deliver gene therapy to Duchenne patients, with the aim of increasing the number of patients who can receive gene therapy treatments.

Several clinical trials are now under way to explore the efficacy of gene transfer, a type of gene therapy, as a treatment for Duchenne. This entails using a micro-dystrophin – a shortened form of the dystrophin gene – with the aim of producing a functional dystrophin protein that patients’ bodies can utilise.

Current investigational gene transfer candidates for Duchenne make use of an adeno-associated virus (AAV). An AAV is a virus that is not known to cause disease and that acts as the delivery vehicle to carry the micro-dystrophin to patients’ cells.

As viral based gene therapies continue to demonstrate therapeutic benefit in the clinic, DRF believes it is crucial to also invest in non-viral delivery methods. These technologies may provide strategies to complement current viral gene therapies by potentially increasing cargo size, widening patient eligibility and potentially offering methods to re-administer gene therapies to patients who have already received a viral-based therapy.

DRF has provided funding for Solid Biosciences’ non-viral delivery research programme, which has partnered with experts across three different labs, two working in nanoparticle formation to develop and optimise a nanoparticle-based gene delivery system, and one working in exosome development as a further alternative to current gene delivery mechanisms.