The therapeutic potential of gene therapy is transformative with the number of gene therapies rapidly increasing. Pharmaron Biologics is a strategic partner for gene therapy developers and operates from state-of-the-art cGMP biomanufacturing facilities in Liverpool. They apply pharma industry-leading expertise and cutting-edge technologies to support customers develop, test and commercialise their innovative medicines. Pharmaron has entered a collaboration with Complement Therapeutics, an early-stage gene therapy company focusing on the development of novel therapeutics for complement-mediated diseases and Cell and Gene Therapy Catapult, an independent innovation and technology organisation supporting gene therapy developers advance faster to clinic to grow the UK economy. The three organisations will combine efforts and expertise, to develop an intensified process for the production of gene therapies, with the aim to increase efficiency while improving the cost-effectiveness of manufacturing. With the growing market demand for AAV products and shift from products targeting small population orphan and rare diseases, towards products targeting high-prevalence and systemic diseases, this collaboration will lay the foundation for the next generation of manufacturing platforms which will be needed to deliver the projected global gene therapy clinical needs, as well as improve patient access to these life-changing therapies.

Six recombinant adeno-associated virus (rAAV) gene therapies have been commercially approved with a growing pipeline of \>550 under development globally. However, they exhibit prohibitive pricing and a long and expensive journey into the clinic partly due to inefficient manufacturing processes. Wider patient access requires increased productivity and efficiency to lower cost-of-goods (COGS), improving affordability to the NHS and healthcare organisations. CGT Catapult is a not-for-profit UK research and technology organisation committed to the advancement of ATMPs and enhancement of the UK industry. CGT Catapult has developed a scalable process and analytical platform for rAAV manufacture and, aligned with its remit to support industry growth, has transferred this platform to leading industry partners and Contract Development Manufacturing Organisations (CDMOs). Touchlight has developed an innovative enzymatic DNA manufacturing platform and GMP production capability which offers the speed, scale and purity to support rapid growth of genetic medicines, substituting plasmid DNA (pDNA) in rAAV production by their proprietary dbDNA. This project aims to combined both assets by the use of dbDNA as a starting material in the CGT Catapult's rAAV manufacturing platform which will enable a more streamlined path to clinical rAAV supply for the UK biotech sector than the current industry standard, pDNA. This will help reduce cost of goods and timelines to access GMP DNA, through utilisation of Touchlight's manufacturing process, and the dissemination and transfer of this dbDNA-based platform to the rAAV industry enabling other UK manufacturing partners to establish their own dbDNA-based manufacturing process thereby improving the successful translation of emerging rAAV based nucleic acid therapeutics from academia to industry. The clinical relevance of this platform will be proven through the supply and use of the Complement Therapeutics rAAV product genome sequence, in connection with the proprietary therapeutic product currently being developed by Complement Therapeutics, in the conduct of the Project by Touchlight, benefiting from accessing this optimised platform through their established relationship with CGT Catapult while reducing their costs and timelines in their journey into the clinic through the use of Touchlight's dbDNA. Touchlight will further characterise rAAV9 production from dbDNA using a new RepCap dbDNA construct. This project will deliver against the UK government's Life Sciences Vision, creating a globally competitive biomanufacturing infrastructure and further establishing the UK's precedence as a leader in innovation, manufacturing and regulation of advanced therapies. This will certainly enable the Next Generation of UK-based Advanced Therapies Innovators and have a big impact in improving rAAV therapies accessibility and affordability bringing them closer to the patients.