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

Abstract

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.

Manufacturing Cell and Gene Therapies: Challenges in Clinical Translation

“…Currently, FDA-approved, commercially available AAV-based therapies target diseases such as retinal dystrophy and spinal atrophy [ 22 ], whereas LV-based gene therapies target diseases such as B-cell lymphoma, β-thalassemia, and cerebral adrenoleukodystrophy [ 23 ]. Both vectors package genes and enable direct administration of the gene of interest to patients [ 24 ]. A major difference between the two types of vectors is that a single DNA strand is packaged in AAVs, whereas an RNA genome is packaged in LVs.…”