About this Research Topic
The emerging field of cell and gene therapies (CGT’s) offers an unprecedented opportunity to treat diseases for which existing interventions are inadequate or simply ineffective. High profile examples such as CAR T-cells and their curative potential for previously terminal illnesses, have fueled excitement among patients, the public, healthcare providers, and investors alike. For CGT’s to fully realise their potential, and live up to high expectations, it will be necessary to overcome many challenges associated with the manufacture and administration of complex ‘living’ therapeutics whilst successfully navigating the regulatory and commercialisation landscapes.
Significant manufacturing gaps (infrastructure and know-how) will emerge if even a handful of CGT products currently in clinical development prove successful, manufacturing costs are high, therapeutic mechanisms of action are often poorly understood and complex, clinical experience is generally limited to a small number of centres, translation of regulations crafted for molecular drugs and biomolecules to CGT’s is often unclear, and a complex IP landscape is evolving. While the field has seen several significant deals with big pharma, hot IPO’s, and biotech’s second largest Series A investment in stem cell therapy start-up BlueRock Therapeutics, these are attached with high expectations for return on investment and aggressive timelines in a yet to be proven arena.
This topic will provide an overview of current state of the CGT industry, critical challenges that need to be addressed ‘now’, anticipate challenges to come, and discuss potential solutions that will enable our pioneers to progress from the frontiers of clinical development to routine therapeutic use. We welcome thought provoking and paradigm challenging manuscripts discussing the many facets of CGT commercialisation and how our nascent industry can realise the fields immense promise to medicine.
Significant manufacturing gaps (infrastructure and know-how) will emerge if even a handful of CGT products currently in clinical development prove successful, manufacturing costs are high, therapeutic mechanisms of action are often poorly understood and complex, clinical experience is generally limited to a small number of centres, translation of regulations crafted for molecular drugs and biomolecules to CGT’s is often unclear, and a complex IP landscape is evolving. While the field has seen several significant deals with big pharma, hot IPO’s, and biotech’s second largest Series A investment in stem cell therapy start-up BlueRock Therapeutics, these are attached with high expectations for return on investment and aggressive timelines in a yet to be proven arena.
This topic will provide an overview of current state of the CGT industry, critical challenges that need to be addressed ‘now’, anticipate challenges to come, and discuss potential solutions that will enable our pioneers to progress from the frontiers of clinical development to routine therapeutic use. We welcome thought provoking and paradigm challenging manuscripts discussing the many facets of CGT commercialisation and how our nascent industry can realise the fields immense promise to medicine.
Keywords: CGT, stem cell processing, gene therapies, CGT products, T-cells
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