Gene and Cell Therapy for Patients

The biopharmaceutical industry is evolving from addressing disease symptoms with drugs administered regularly over time, to potentially curing diseases in as little as a single administration of a therapy. Gene and cell therapies are leading this movement, where a combination of engineered cells or genes are used to modify or repair diseased or damaged tissues, immune cells, or DNA and RNA.

The development of these therapies has been made possible with a tremendous investment of time and money by academia and the biopharmaceutical industry into further understanding biology and the advent of tools to selectively repair or disable defective genes in patients.

Today, the first wave of gene and cell therapies have been approved with another 40 gene and cell therapies in mid to late-stage clinical development, all for the treatment of rare and debilitating diseases.

A primary challenge to providing cell and gene therapies to many patients is that the healthcare system is set up to reimburse relatively lower-cost therapies administered over time. Gene and cell therapies conversely involve a high upfront cost for a treatment that might be administered only one time. As a result, there has to be a shift in how costs are managed and potentially transferred from one payer to the next.  These new therapies, while costly upfront, are expected to show a tremendous cost-benefit over time, resulting in tremendous savings for the healthcare system, and lead to a greater quality of life in patients.