Innovation in gene therapy brings the potential for transforming patient care and obviating the need for chronic therapy through single-dose cures. Despite the potential long-term benefits of this new therapeutic modality, gene therapy companies face a number of underappreciated challenges.
Over the past four decades, and especially in recent years, progress in the development of cell and gene therapies has reached unprecedented levels. In gene therapy alone, between 1989 and 2015 there were more than 2,330 clinical research programs targeting almost 50 different indications.1,2 As more cell and gene therapies reach commercial stage, industry insiders expect research in the sector to continue to expand in the coming years. For patients, clinicians, and health systems, new cell and gene therapies bring the promise of historic and transformative advances in the treatment of many serious diseases.
Streamlining, connecting, and automating workflows to shape the future of cellular treatment delivery has the potential to transform how we treat and potentially cure once life-threatening diseases.
Emerging cellular therapies aim to manufacture cell populations for clinical indications, such as cancer, autoimmune, and cardiovascular diseases. As hospitals and translational facilities explore implementation of this technology they concurrently evaluate whether or not to manufacture the therapies onsite. Here is a guideline of how and when to get started.
Because the parameters for ex vivo cell and gene therapy manufacturing differ considerably from chemical drug production, life sciences companies are likely to encounter challenges along key stages of the product life cycle.
Through proper implementation of ICH Q12 tools and enablers, the industry could manage CMC changes effectively under a company’s pharma quality system with less need for oversight prior to implementation.
This article outlines six recent regulatory changes in China intended to protect patient safety as well as reduce regulatory burdens and minimize delays.
In today’s changing industry, it is critical we consider several factors about cell culture bioprocessing and the impact they will have on the future of patient treatment.
As the second wave of CAR-T therapies begins coming to market in the next five years, their manufacturers will need to anticipate and prepare to address many of the same commercial challenges as their predecessors, as well as several additional ones.
Gene therapy may be the next revolution in healthcare. The opportunity to cure diseases through the modification of the genome was once considered science fiction, but no longer. The United States has seen a few approvals in this area, including the approval of voretigene neparvovec for a rare form of blindness. In addition, CAR-T agents, two of which are available, have demonstrated alternative ways to treat disease by manipulating genes of different cells.