Report on Webinar Series Cell and Gene Therapy: From Concept to Clinical Use

With the launch of the UK Academy of Pharmaceutical Sciences Advanced Therapy Medicinal Products Focus Group in late 2020, a webinar series reviewing the current and emerging trends in cell and gene therapy was held virtually in May 2021. This webinar series was timely given the recent withdrawal of the United Kingdom from the European Union and the global COVID-19 pandemic impacting all sectors of the pharmaceutical sciences research landscape globally and in the UK. Delegates from the academic, industry, regulatory and NHS sectors attended the session where challenges and opportunities in the development and clinical implementation of cell and gene therapies were discussed. Globally, the cell and gene therapy market has reached a value of 4.3 billion dollars in 2020, having increased at a compound annual growth rate of 25.5% since 2015. This webinar series captured all the major developments in this rapidly evolving area and highlighted emerging concepts warranting cross-sector efforts from across the community in the future.

Electro-mechanical transfection for non-viral primary immune cell engineering

Non-viral approaches to transfection have emerged a viable option for gene transfer. Electro-mechanical transfection involving use of electric fields coupled with high fluid flow rates is a scalable strategy for cell therapy development and manufacturing. Unlike purely electric field-based or mechanical-based delivery methods, the combined effects result in delivery of genetic material at high efficiencies and low toxicity. This study focuses on delivery of reporter mRNA to show electro-mechanical transfection can be used successfully in human T cells. Rapid optimization of delivery to T cells was observed with efficiency over 90% and viability over 80%. Confirmation of optimized electro-mechanical transfection parameters was assessed in multiple use cases including a 50-fold scale up demonstration. Transcriptome and ontology analysis show that delivery, via electro-mechanical transfection, does not result in gene dysregulation. This study demonstrates that non-viral electro-mechanical transfection is an efficient and scalable method for cell and gene therapy engineering and development.

Developing gene therapies for rare diseases: an interview with Geoff MacKay

Geoff MacKay is a pioneer in cell and gene therapy with a track record of successful leadership at innovative biotechs. He is currently the president and CEO of AVROBIO, a clinical-stage lentiviral gene therapy company that treats lysosomal disorders, and a board member of Talaris Therapeutics and Satellos Bioscience. He is also the founding CEO of eGenesis, a biotech that applies CRISPR-Cas9 gene editing to xenotransplantation and the former president and CEO of Organogenesis, a world-leading cell therapy company. Earlier in his career, MacKay spent 11 years at Novartis in senior leadership positions within the global transplantation and immunology franchise.