12/29/2023 0 Comments Juno ds editor![]() Targeting of specific tissues is required for the successful delivery of genetic materials with in vivo therapies. In vivo ATMPs include the use of alternative gene delivery systems that may be viral (e.g., adeno-associated virus or lentiviral) or non-viral (e.g., polymeric and lipid nanoparticles) in composition. Allogenic cell therapies exemplified by mesenchymal stem cells or pluripotent stem cells can be manufactured at a commercial scale and are available as off the shelf products. This is followed by infusion of the genetically altered autologous cells to elicit a therapeutic response. Cell therapies may be broadly referred to as autologous cell therapies in which direct isolation of cells from patients, their culture, expansion and genetic modification takes place outside the body. ![]() The majority of ATMPs under investigation in the UK are based on viral vectors, with the portfolio of therapies under investigation showing equal representation of both ex vivo and in vivo gene therapies.Įx vivo strategies for genetic alteration include cell therapies with a rapidly growing global market predicted to reach 45.4 billion dollars by 2028. In 2020, 154 ATMP clinical trials were documented in the UK alone, representing a 12% global share of ongoing ATMP trials and evidence of UK leadership in this area. As of October 2021, there are >20 cell and gene therapy products that are licensed from the FDA Office of Tissues and Advanced Therapies (OTAT). The global ATMP market is a rapidly growing sector, reaching a market worth of 7.9 billion dollars in 2020. ![]() The session was attended by delegates representing the academic, pharmaceutical industry, pharmacy practice and regulatory sectors a diversity which was reflected in the speaker line-up and panel discussion. This was a flagship event for the Advanced Therapy Medicinal Products (ATMP) Focus Group which was launched in fall 2020. The Academy of Pharmaceutical Sciences (APS- the UK Pharmaceutical sciences community) hosted a virtual webinar series in May 2021 on the topic of cell and gene therapy, holistically combining concepts from bench discovery to their clinical implementation. ![]() 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. 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. 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. 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. 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.
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