Progress in development of gene and cell therapies around the world has potential to transform standards of care for a range of diseases and address significant areas of unmet need in healthcare over the coming years. In the U.S. alone, almost 20 gene and cell therapy products have been approved thus far,1 with many other development programs reaching later clinical stages. The technology platforms of many of these drugs also offer the potential for curative efficacy and expansion for use in multiple indications.
The regenerative medicine sector is at a remarkable moment. Transformative products are now on the market and accessible to greater numbers of patients every day. Dozens of additional therapies are in late stage studies. The regulatory and policy environment has evolved rapidly alongside the science, enabling a surge of incoming innovation.
A number of challenges are hindering efforts to develop cell and gene therapies to treat rare diseases. While these challenges may seem common across other drug markets, in the case of rare diseases, they are exacerbated by limited patient populations
According to a review of recent research studies that measured U.S. public opinion related to gene medicine, the public is not yet aware of gene medicine, is unfamiliar with the terms being used to describe the topic, and, when faced with multiple therapeutic options, is worried about making informed decisions.
Current reimbursement models in general do not accommodate many of the unique factors that are common among gene and cell therapies.
Manufacturing viral vector-based therapies such as vaccines and gene and cell therapies is complex, but a new manufacturing solution helps solve those challenges.
Cell and gene therapies: Will their use become ubiquitous, changing the landscape forever as many have promised? Or will applications be confined to rare diseases as they have been thus far?
Despite recent progress in regenerative medicine, cell and gene therapy executives are still pioneering many new pathways as they shape science into never-before-realized medicinal applications. From innovation and growth come surmountable, yet challenging, issues which can hinder — even halt — groundbreaking progress in its tracks.
A range of factors — including small patient populations, complex manufacturing processes, and lack of specialized expertise — are positioned to both drive up costs and require new options for stakeholder engagement and risk sharing along the development pathway. New approaches in development are needed to support the next generation of novel drugs on the horizon.
Single use technologies are expected to play a big role in the commercialization of cell and gene therapies because of their cost, speed, and sterility benefits.