TherageniX and UK Uni team to develop powdered gene therapy

Innovate UK has awarded TherageniX and the University of Nottingham $1.2 million to develop a powder non-viral gene delivery system for bone regeneration.

TherageniX, a spin-out from the University of Nottingham, is developing a dry powder gene therapy formulation that aims to be used for bone graft augmentation. Innovate UK has provided TherageniX and its collaborator, the University of Nottingham, with a £995,000 ($1.2 million) grant. The funding will support the development of the gene delivery system to improve the outcomes for patients undertaking bone grafting procedures.

Initially, the grant will focus on orthopaedic applications and Anandkumar Nandakumar, CEO of TherageniX, told BioProcess Insider the funding will “allow us to test different manufacturing methods and we will select the optimal method based on factors such as cost [and] stability.”

DepositPhotos/j.dudzinski

While TherageniX acknowledged the transplantation of autologous bone “is the gold standard bone repaid strategy,” the company said it has “drawbacks” and many bone implants risk failing because of poor integration or infection. In turn, patients can experience delayed recovery, re-operation, and higher costs of treatment.

TherageniX’s non-viral gene delivery system transforms a liquid formulation into a dry powder gene therapy. It works by combining autologous bone marrow cells from the individual and the firm’s platform technology then advances the production of genes to aid improvement of the regenerative capacity of bone, skin, muscle, and cartilage post-surgery. Furthermore, the quick transfection of a patient’s cells results in no additional time needed in the operating theater.

The grant will run for over two years and the two parties will hire a team to work on the project, however a specific headcount has not been disclosed. Additionally, the work will take place at the University of Nottingham’s laboratories with help from various partners based in the UK.

Dry powder benefits

The handful of gene therapies available in the space are single-dose injections or infusions, but the US Food and Drug Administration (FDA) approved the first ‘redosable’ gene therapy in May, in the form of Krystal Biotech’s gel, Vyjuvek.

With non-traditional methods of gene therapies receiving approval and grants, Nandakumar said “different ways of administration are one of the facets of how the field is evolving.”

The benefits of a dry powdered gene therapy include: “Simpler storage so that highly specialized equipment may not be needed, which also means that we can deploy it in areas that do not have access to such equipment. A longer shelf life that allows the end user to stockpile and us, as manufacturers, to have larger batch sizes to reduce costs. It is also easier to handle a powder compared to liquids during transportation.”