Gene therapy CMC presents unique challenges

Chemistry, manufacturing, and controls (CMC) is an integral part of the production of any pharmaceutical product. The intricacies and rapid advancement within the gene therapy space have created unique challenges to the CMC process including meeting development needs, global regulatory compliance, and timelines. To optimize the production of our pipeline therapeutics in a timely fashion and ensure scalability and consistency commercially, we have developed a customized approach to CMC.

Product purity through process purity

Our CMC strategy is optimized to produce high-quality clinical material in support of current and future indications using cutting-edge production systems to maximize both vector yield and purity. Control of the process and product impurity profile guarantees a highly efficacious and safe outcome. Our strategy involves building a commercial process utilizing “quality by design” and “manufacturing by design” principles to eliminate future roadblocks during process validation and product approval.