Bitrobius Genetics, a genetic medicines startup based in Macclesfield, has raised £700,000 in grant funding from the UK's Advanced Research + Invention Agency (ARIA). The award funds a collaboration with the Medical Research Council Mitochondrial Biology Unit (MRC MBU) of the University of Cambridge to apply Bitrobius' core technology to the genetic modification of mitochondria.
At the centre of that technology is Gentrafix™, a platform that aims to use plasmid DNA to replicate within human cells and then travel to adjacent cells, spreading the therapy efficiently throughout affected cells. Current gene therapy approaches are only able to treat a small proportion of affected cells.
Most genetic diseases are caused by mutations in genes, most commonly in the nucleus of cells, and gene therapy treats these diseases by delivering a functional copy of the affected gene. Mitochondria, the tiny structures within cells that generate much of the energy needed for life, also contain their own small genome, and mutations in this mitochondrial DNA cause a range of severe inherited diseases for which treatment options remain extremely limited. Delivering DNA into mitochondria remains a major unsolved challenge because their double membrane forms a significant barrier. Overcoming that barrier could have far-reaching implications, as mitochondrial dysfunction is associated not only with inherited mitochondrial diseases but also with major health challenges including neurodegenerative disorders such as Alzheimer's and Parkinson's, cancer, diabetes and ageing.
The ARIA grant will enable Bitrobius to broaden the application of Gentrafix™ to solve the problem of gene delivery to mitochondria, delivering it across a double mitochondrial membrane using a proprietary DNA-secreting pore. Backing to date includes seed investment from Science Creates Ventures (SCVC), Future Planet Capital, Synbioven and Catapult Ventures, plus grant funding from Innovate UK.
We are excited to receive the funding to apply our groundbreaking technology to the ambitious challenge of DNA delivery to mitochondria, thanks to the support of ARIA and the Mitochondrial Biology Unit at the University of Cambridge.
We are excited to combine Bitrobius' innovative technology with our expertise in mitochondrial genetics to tackle one of the field's most longstanding challenges: delivering DNA into mitochondria.
