Development of an Innovative Supply Chain Model to Expedite Delivery of Viral Vector ATIMPs to Clinical Trials

"The rapid development of novel treatments called Advanced Therapies, such as cell therapies and gene therapies have required new manufacturing facilities and technologies worldwide. Manufacturing of gene therapy viral vectors for human use requires specialised high-quality manufacturing facilities and there is currently insufficient such capacity in the UK and globally. Reliance on overseas CMOs presents challenges in terms of both capacity and lack of flexibility. The Cell and Gene Therapy Catapult has recently opened its manufacturing centre in Stevenage offering an innovative cost-sharing model for small gene therapy companies, such as Freeline, to establish an internal manufacturing capability for their drug substance. The aim of this project is to further develop the UK based supply chain by establishing an integrated, lean partnership between CGTC-MC and Symbiosis, a UK company that specialises in drug product manufacture (fill finish) for clinical sterile drug products and small volume, niche commercial sterile products.

This collaboration, between Freeline, CGTC-MC and Symbiosis aims to establish an efficient, secure supply chain to reliably deliver viral vector gene therapy drug products to clinical trials. This will speed the pace at which new gene therapies can be made available to patients. The clinical supply chain delivered by this project will provide a foundation on which to deliver commercial supplies of gene therapy products in the future, thus establishing a secure UK-embedded commercial supply chain.

The three UK collaborators will work together to create the new processes and analytical technologies to achieve rapid and seamless transfer of materials between the partners whilst maintaining quality. The project will include alignment of respective quality systems and processes as well as investment in internalising critical quality control tests and establishing new novel rapid methods. Internalising QC will bring about process efficiencies, adding to the security of the supply chain.

The new integrated operating model will be demonstrated with the manufacture and supply of a GMP batch of a Freeline gene therapy drug product to a Phase I/II clinical trial for the treatment of a lysosomal storage disorder. The resulting supply chain and technologies will be translated into standard processes which can be offered by CGT-MC and Symbiosis to other future viral vector customers, thus significantly enhancing the UK viral vector manufacturing capability"