When we think about cell and gene therapies (CGTs) what most often comes to mind is the early stage scientific innovation responsible for finding new ways to treat diseases. A lot of complex research and testing is performed to confirm Proof of Concept and that the therapy performs as desired.
Congratulations! That is a huge accomplishment.
The next step is to prepare the product for in-human testing. Clinical protocols are developed in consultation with the regulatory authorities, a target patient population is identified, clinical investigators and an appropriate contract research organization are selected, and funds are obtained to begin the studies. What is sometimes overlooked, or left to the last minute, is the less-than-glamorous, but still critical, manufacturing steps that ensure a new, potentially life-saving treatment reaches patients in a safe and reliable form.
In this post we will map the five critical steps that require thoughtful measured consideration for a CGT product to be produced in a good manufacturing practices (GMP) facility.