TherageniX, U.K. University Developing Bone Regeneration Therapy

TherageniX, a University of Nottingham spin out pioneering a dry powder gene therapy formulation for bone graft augmentation, and the University of Nottingham have been awarded a £995,000 grant from Innovate U.K. to develop the company’s gene therapy in a powder, a non-viral gene delivery system to improve tissue regeneration after surgery. TherageniX will initially focus on orthopaedic applications, with the aim of improving patient outcomes in bone grafting procedures.
 
TherageniX’s proprietary technology allows rapid transfection of patients’ cells with the gene(s) of interest. Following this intra-operative procedure, the transfected cells are then implanted at the surgical site, enabling the body to produce the relevant proteins or factors for tissue regeneration. The grant awarded in response to Innovate U.K.’s call for “innovative technologies for intracellular drug delivery” as part of its Transforming Medicines Manufacturing program, will allow the company to focus on developing its non-viral gene delivery system, transforming its liquid formulation into an enhanced dry powder gene therapy.

“Our mission is to advance treatments in repairing tissue damage and we are grateful for the support from Innovate U.K. at this early stage. This grant is testament to the potential of TherageniX’s novel approach to tissue regeneration and will enable us to make great strides in manufacturing our powdered gene therapy,” TherageniX CEO Dr. Anandkumar Nandakumar said. “We aim to accelerate our R&D pipeline and strengthen our team and hope to continue actively engaging prestigious funding bodies and investors that will enable us to deliver an improved solution to bone grafting that will help patients, physicians, and payors.”
 
While any tissue can be targeted using the patented technology, TherageniX will focus orthopedic applications first. It will combine autologous bone marrow cells from the patient with its platform technology to drive the production of genes, such as BMP for osteogenesis, helping to improve the regenerative capacity of skin, bone, muscle, and cartilage following surgery. 
 
The transplantation of autologous bone is the gold standard bone repair strategy. However, this process has drawbacks and bone implants may fail due to poor integration or infection at the harvest site, or do not provide satisfactory functionality. This can result in delayed recovery, re-operation, poor quality of life and increased costs.¹ TherageniX’s gene therapy technology² improves upon these bone-grafting procedures, using the body’s cellular machinery to enhance regeneration at the surgical site. The technology easily integrates with established surgical procedures, and rapid transfection of patient’s cells means no additional time is required in the operating theatre. It offers a simple and cost-effective solution to help improve regeneration, leading to better functional outcomes for patients. 
 
“Adapting technology for a rapid application directly to grafted tissue within the operating theatre has been a vision for our gene delivery platform for several years. We have the opportunity here to bring regenerative medicine and gene therapy forward with innovative applications and apply it in ways we could not have envisaged only a few years ago, even to emergency medicine,” noted Dr. James Dixon, associate professor of the School of Pharmacy and NIHR Nottingham Biomedical Research Centre at University of Nottingham, and TherageniX platform creator. “Deployment via a dry powder will allow us to overcome some of the bottlenecks for its impact. We hope that our system will generate a platform of transformative, and economically viable approaches to clinical problems that remain poorly addressed in modern medicine.”
 
TherageniX, a healthcare startup built by NLC, the European healthtech venture builder, was incorporated in 2022 with the support of Nottingham Technology Ventures Ltd. 

References
¹ https://doi.org/10.1016/j.actbio.2010.07.035; https://doi.org/10.1186/1749-799X-8-33
² https://doi.org/10.1016/j.biomaterials.2019.119277