Non-viral delivery for gene therapyA next generation gene therapy delivery approach
The Duchenne Research Fund has committed £640,000 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.
We look forward to sharing further news about this exciting next generation gene therapy approach in due course.
Cambridge, MA, USA
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We are thrilled that the money raised through the Duchenne Research Fund’s 2013 dinner and donations since then have enabled us to seed fund the incredible Solid GT project, which has today received industry investment (read the press release below). While gene therapy is an exciting piece of the puzzle for beating Duchenne for all boys, it is now more critical than ever that we continue to raise vital funds to push Duchenne therapies into clinical trial as quickly and safely as possible and to get them to our boys NOW.
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