The FDA’s approval in December of a new gene editing treatment for sickle cell disease not only represents a potential cure for a painful illness that afflicts millions of people globally but could be a harbinger of a surge in cell and gene therapy (CGT) activity throughout 2024 and beyond. This prediction is supported by a report from the American Society of Gene and Cell Therapy that as of the third quarter of 2023, there were 3,866 therapies in various stages of development. McKinsey has projected that there will be 22 cell therapy and as many as 31 gene therapy launches in 2024.
As promising as all that activity is for public health and CGT market players, it exposes a potentially serious issue with patient and provider access—one that threatens to complicate providers’ work, delay patient access to treatments, and hinder overall market development. This threat isn’t from the therapies themselves, but rather from the lack of standardization across the platforms that patients and providers use for onboarding and coordinating patient treatment. Today there seem to be as many access portals as there are commercially available CGT treatments, with each portal having its own unique processes and requirements.
If cell and gene therapies are going to be fully scalable and fulfill their market potential—Vantage Market Research projects the global CGT market will grow at an average annual rate of 18.2% this decade —then the focus must be on delivering a standardized us.