Bitrobius wins GBP £700,000 to target mitochondria

Bitrobius Genetics and the University of Cambridge have secured a £700,000 grant from ARIA to develop mitochondrial gene delivery technology. The funding will support work on getting DNA across the mitochondria's double membrane.

The project brings together Bitrobius and the Medical Research Council Mitochondrial Biology Unit at the University of Cambridge to address a long-standing problem in mitochondrial medicine. Researchers have struggled to deliver genetic material into mitochondria because the structures are enclosed by two membranes.

Mitochondria carry their own small genome, separate from the DNA in the cell nucleus. Mutations in mitochondrial DNA can cause severe inherited disorders, and treatment options remain limited because faulty genes inside the organelle are difficult to replace or repair.

The grant will support efforts to adapt Bitrobius's Gentrafix platform for mitochondrial use. The company is developing the system around plasmid DNA that it says can replicate within human cells and move into neighbouring cells, unlike existing gene therapy approaches that often reach only a fraction of affected cells.

Under the programme, Bitrobius aims to use what it describes as a proprietary DNA-secreting pore to move DNA across the mitochondrial double membrane. If successful, the approach would address one of the main technical obstacles in mitochondrial gene therapy.

The delivery barrier

The challenge has wider scientific interest because mitochondrial dysfunction is linked not only to inherited mitochondrial disease but also to a range of other disorders. Researchers have associated defects in mitochondrial function with neurodegenerative conditions including Alzheimer's and Parkinson's disease, as well as cancer, diabetes and ageing.

Even so, the immediate focus is inherited disease caused by mutations in mitochondrial DNA. Gene therapy has advanced in disorders linked to nuclear genes, but translating that progress to the mitochondrial genome has proved harder because the target sits behind an additional membrane barrier.

ARIA, the UK's Advanced Research and Invention Agency, was established to fund high-risk research programmes. This award places the Bitrobius-Cambridge collaboration within that remit, targeting a problem that has resisted conventional approaches.

Bitrobius is a UK genetic medicines company backed by seed investment from Science Creates Ventures, Future Planet Capital, Synbioven and Catapult Ventures. It has also received grant support from Innovate UK.

The partnership combines delivery technology with specialist mitochondrial research.

"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," said Dr Rocky Cranenburgh, chief executive officer and founder of Bitrobius Genetics.

The academic side of the collaboration comes from the MRC Mitochondrial Biology Unit, which specialises in the genetics and biology of mitochondria. That expertise is expected to complement Bitrobius's work on DNA delivery systems and cell-to-cell spread.

The effort reflects a broader push in genetic medicine to improve how therapies are delivered after a gene target has been identified. For many rare inherited diseases, the scientific hurdle is no longer only which gene to replace, but how to reach enough of the right cells and get the therapeutic material into the right part of the cell.

In mitochondrial disease, that challenge is more acute because therapeutic DNA must cross two membranes instead of one. Researchers have spent years trying to solve that problem, making delivery, rather than gene design itself, the main bottleneck for progress in the field.

"We are excited to combine Bitrobius's innovative technology with our expertise in mitochondrial genetics to tackle one of the field's longest-standing challenges: delivering DNA into mitochondria," said Minczuk.