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 Duchenne Research Fund is delighted to announce that we are awarding a £640,000 grant to fund vital psychosocial care and research as part of Duchenne UK’s DMD Care UK programme.
We are pleased to share that Vamorolone, an alternative medicine to corticosteroids, has shown positive results in a Phase 2 Study …
Watch the highlights of Gavriel Rosenfeld in conversation with Dr Ronni Cohn at our virtual Evening with the Duchenne Research Fund. More than 500 people watched our virtual event to hear Gavriel talk about living with Duchenne, mental health, juggling school and hospital visits, and more …
Learn more about what we fund.
Find out how you can help us beat Duchenne.