Bitrobius targets mitochondrial gene delivery with £700k ARIA grant
Bitrobius Genetics has been awarded a £700,000 grant from the Advanced Research + Invention Agency (ARIA) to investigate whether its gene-delivery technology can be adapted to deliver DNA into mitochondria.
The project will be carried out in collaboration with the Medical Research Council Mitochondrial Biology Unit (MRC MBU) at the University of Cambridge, bringing Bitrobius' Gentrafix platform together with expertise in mitochondrial genetics.
Most genetic diseases are caused by mutations in genes located in the cell nucleus, and gene therapies typically work by delivering a functional copy of the affected gene to cells. Mitochondria also contain their own genome, however, and mutations in mitochondrial DNA can cause severe inherited diseases for which treatment options remain limited.
A major challenge is getting DNA into mitochondria as the organelles are surrounded by a double membrane that presents a significant barrier to delivery.
Bitrobius is developing Gentrafix, a platform based on plasmid DNA designed to replicate within human cells and subsequently move into adjacent cells. The company says this could allow genetic material to reach a greater proportion of affected cells than approaches that deliver therapy to individual cells.
Itse new project will investigate whether Gentrafix can be adapted to deliver DNA across the mitochondrial double membrane using a proprietary DNA-secreting pore.
“We are excited to receive the funding to apply our groundbreaking technology to the ambitious challenge of DNA delivery to mitochondria,” said Dr Rocky Cranenburgh, CEO and founder of Bitrobius Genetics.
The collaboration will combine development of the delivery technology with research into mitochondrial genetics 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,” said Professor Michal Minczuk, Professor of Mitochondrial Genetics at the University of Cambridge.
Mitochondrial dysfunction is associated with a range of diseases and conditions, including inherited mitochondrial disorders, neurodegenerative diseases, cancer, diabetes and ageing. However, the immediate focus of the project is the technical challenge of mitochondrial DNA delivery rather than demonstrating therapeutic efficacy in these conditions.
The ARIA-funded work will therefore test whether Bitrobius' existing gene-delivery approach can be extended to a different cellular target, potentially opening a route towards genetic medicines for diseases involving mitochondrial DNA. Pic: NCI
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