Cell and Gene Therapy

Emerging cell and gene therapies offer innovative approaches to treating disorders with a high unmet need. Advanced Therapy Medicinal Products (ATMPs) provide targeted and effective treatments for both inherited disorders and complex acquired conditions including various forms of cancer and degenerative diseases.

What challenges will be addressed?

1. Delivery of therapeutic genes or gene editing tools is the rate limiting aspect of ATMP development. How can delivery be enhanced using existing or novel delivery technologies?
2. Synthetic evolution approaches have revolutionised our understanding of host-viral vector interactions. Can analogous evolutionary/selection methods be used to enhance gene editing approaches and/or cell therapy payloads?
3. How can we utilise artificial intelligence/machine learning to speed ATMP development?

how will collaboration enable success?

Research collaborations will be driven by the shared application of advanced gene transfer methods and tools. There are two technological areas that will help drive collaboration:

Much of the explosion in in vivo gene delivery has been driven by methodologies that allow the creation of novel rAAV serotypes and their evolution, selection, and refinement.  We will share best practices, methods and suitable libraries in order to stimulate collaborations aimed at enhancing novel serotype identification and use.

Many of the advances in the gene editing space have been driven by methodologies that allow the selection of novel protein activities. The emergence of bacteriophage-driven PACE technology has democratised the creation and optimisation of novel protein/protein and protein/nucleic acid interactions important for ATMP generation. We will share best practices, methods and appropriate plasmid constructs to facilitate the creation of novel protein-based IP that supports ATMP development.

By initially focusing on these two technologies, we will catalyse new collaborative initiatives to quickly and inexpensively generate novel ATMPs.