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
Latest research news
Santhera Pharmaceuticals has announced that partner ReveraGen Biopharma has completed a long-term, open-label extension study of 24 months duration with Vamorolone in patients with Duchenne…
Solid Biosciences has issued an update on IGNITE DMD, the Phase I/II clinical study with its microdystrophin gene therapy, SGT-001.
The Duchenne Research Fund is pleased to share that the ground-breaking KineDMD activity monitoring study at Imperial College London/GOSH has been extended into 2021…
The Duchenne Research Fund has awarded a grant to Dr Barry Byrne and Dr Manuela Corti’s team at the University of Florida, whose vital work focuses on overcoming immunological challenges to ensure all boys and young men can benefit from gene therapy treatments for...
We are delighted to share the news that Dr Ronald Cohn, the head of the DRF’s Scientific Advisory Board, has been made President and CEO of the Hospital for Sick Children …
Solid Biosciences announces preliminary gene therapy clinical trial results and intention to dose escalate
Solid Biosciences has announced preliminary data from its gene therapy clinical trial, IGNITE DMD …
Learn more about the Duchenne Research Fund.
Learn more about what we fund.