The Cell and Gene Therapy Catapult (CGT Catapult), an independent innovation and technology organisation specialising in the cell and gene therapy industry; Pharmaron Biologics (UK) Ltd, a strategic partner for gene therapy developers; and Complement Therapeutics, a preclinical stage biotechnology company developing novel therapeutics for complement-mediated diseases, have been awarded a £1.4m Innovate UK Transforming Medicines Manufacturing (TMM) Programme grant to develop a perfusion process that will improve gene therapy production.
Adeno-associated virus (AAV) vectors are used in a number of approved gene therapies that target rare conditions. Building on the success of these therapies, the sector is now developing AAV-based gene therapies that aim to target higher-prevalence conditions.
The CGT Catapult, Pharmaron Biologics (UK) Ltd and Complement Therapeutics will collaborate in a two-year project to develop an intensified perfusion process for the production of AAV. Perfusion processing enables the continuous exchange of fresh culture medium and the removal of waste. This allows for AAV to be produced from a higher density of cells, over extended periods of time, reducing production costs.
As the lead applicant, Pharmaron Biologics (UK) Ltd will apply the process to its AAV platform, helping gene therapy developers that it works with to benefit from improvements in AAV yield, quality and lower manufacturing costs.
Complement Therapeutics aims to harness learnings from the new process for future development of its portfolio of treatments for complement-related diseases each with a high unmet clinical need.
Matthew Durdy, Chief Executive of the Cell and Gene Therapy Catapult, said: “AAV is a crucial element in many gene therapies yet there are currently no clinically approved AAV products manufactured using perfusion technologies. Working with experts at Pharmaron and Complement Therapeutics, this project offers a valuable opportunity to unlock the benefits of this manufacturing process. This aims to help make gene therapies more affordable to healthcare systems and ensure that sufficient volumes of AAV can be produced to meet an increase in demand.”
Derek Ellison PhD, VP Biologics Services Europe at Pharmaron Biologics (UK) Ltd, said: “While perfusion has been successfully applied to intensify production and improve manufacturability and process economics for more traditional biologics such as recombinant proteins, there are few, if any, successful applications for gene therapies. Leveraging the advanced processing and product characterisation capabilities of Pharmaron’s Liverpool site, allied with the technologies and expertise of Complement Therapeutics and the CGT Catapult, this project aims to make a major breakthrough in this area, to the benefit of patients and customers.”
Rafiq Hasan, Chief Executive Officer of Complement Therapeutics, said: “We are excited to collaborate with Pharmaron and the CGT Catapult on this project. Developing a perfusion process for AAV production holds significant promise for advancing our pipeline of treatments for complement-mediated diseases. This initiative not only represents a technological leap forward but also aligns with our mission to address high unmet clinical needs and ultimately improve patient outcomes.”
