Gene editing therapy start-up secures £3.4 million grant for DMD research
Biotech company MyoGene Bio have received a grant from the California Institute of Regenerative Medicine to study innovative gene-editing therapy, thanks to support from Duchenne UK in collaboration with other charities.
Gene editing is a ground-breaking approach to treating genetic disorders such as Duchenne muscular dystrophy (DMD). It aims to correct faulty genes by altering patients’ DNA sequences. It differs from other forms of gene therapy, which aim to deliver new genetic material into cells.
The research, which uses a revolutionary technology called CRISPR to precisely cut and replace DNA, will be tested in animal models of DMD. If it is shown to be safe and effective, MyoGene hope to apply for Investigational New Drug status with the US Food and Drugs Administration (FDA), so that it can be tested in humans by 2024.
Duchenne UK co-funded MyoGene’s grant application alongside the National Institute of Arthritis and Musculoskeletal and Skin Diseases and Parent Project Muscular Dystrophy, helping to advance this essential pre-clinical research.
Duchenne UK Director of Research, Alessandra Gaeta, says: “We are proud to support this innovative research which has the potential to be transformative for DMD patients across the world. By enabling early studies into gene editing, we are helping to accelerate its development and paving the way for the therapy to arrive in clinics. ”