Non-viral delivery for gene therapyA next generation gene therapy delivery approach
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.
Cambridge, MA, USA
Latest research news
The FDA has granted accelerated approval for the gene therapy treatment SRP-9001. This is the first time a gene therapy for Duchenne has been approved anywhere in the world …
On Rare Disease Day, watch Eli Crossley, son of our friend and Duchenne UK co-founder Emily, as he talks about – and demonstrates – the amazing technological developments that are being trialled for children with Duchenne muscular dystrophy. The ‘smart suit’ aims to help children who are losing upper body function in still continuing to use their arms.
We are delighted that the ground-breaking KineDMD activity monitoring study we funded has been published in the prestigious scientific journal Nature Medicine, and covered by the BBC …
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