Business Engagement Fund award to support mitochondrial disease research
Stealth BioTherapeutics, a commercial-stage biotechnology company focused on the discovery, development, and commercialisation of novel therapies for diseases involving mitochondrial dysfunction, have today announced an award from the Network’s Business Engagement Fund to support a new collaborative research project involving the Mary Lyon Centre at MRC Harwell (MLC), the MRC National Mouse Genetics Network (NMGN), and researchers at University College London (UCL).
The aim of this collaboration is to evaluate the effects of SBT-589, one of Stealth BioTherapeutics’ lead pipeline compounds, on survival rates, motor function, and biomarkers of disease in a mouse model of Leigh syndrome. Mitochondrial dysfunction is central to Leigh syndrome pathology, affecting cells that supply energy to the brain, nerves, and muscles. Previous preclinical studies have shown that SBT-589 improved bioenergetics in Leigh syndrome patient-derived cells, and ameliorated pathology associated with mitochondrial dysfunction and neurodegeneration across animal models of disease.
This award builds on Stealth BioTherapeutics’ established collaboration with investigators within the Network’s Mitochondria Cluster, including Professor Robert Pitceathly of UCL, and on prior clinical research in primary mitochondrial myopathy including multiple Mitochondria Cluster members.
“I’m excited to support the preclinical evaluation of SBT-589, leveraging our NMGN MitoCluster platform at the MLC to deeply characterise mitochondrial dysfunction in disease models. Combined with the extensive clinical and non-clinical expertise in mitochondrial pathology contained within our consortium, we aim to accelerate the development of therapeutics, such as SBT-589, towards clinical applications,” said Professor Pitceathly.
The Network’s Business Engagement Fund is designed to foster meaningful partnerships between industry and the global research community, accelerating the translation of scientific discovery into real-world patient impact.
“This collaboration represents an important step forward for our pipeline and our long-standing engagement with the mitochondrial research community,” said David A. Brown, Ph.D., Stealth BioTherapeutics’ Chief Scientific Officer. “Testing promising assets in disease-relevant animal models has historically been a barrier to progress in mitochondrial drug development. This project helps address that gap and strengthens our ability to translate innovative science and advance potential therapies into the clinic.”
“The Business Engagement Fund is designed to bring the expertise of the MRC National Mouse Genetics Network together with the innovation capacity of industry partners. By supporting early-stage collaborations, we can help accelerate the translation of discoveries from advanced model systems into new approaches for understanding and treating disease, including in important areas such as mitochondrial biology,” said Professor Owen Sansom, Director of the MRC National Mouse Genetics Network.
By strengthening ties between industry and academia, the project is expected to accelerate the progression of promising mitochondrial therapies toward clinical development and future regulatory milestones, supporting shared goals to address the significant unmet needs faced by individuals living with diseases of mitochondrial dysfunction.