CMC – Gene Therapy’s “Achilles Heel”

Patient access to novel gene therapies is often obstructed by manufacturing challenges, in particular, issues with Chemical, Manufacturing and Controls (CMC). With increasing scrutiny from health technology assessment (HTA) agencies, biopharma companies must establish excellent and transparent CMC processes to be successful. In a recent article in Cell and Gene, Andrew Knudten, Chief Operating Officer of Jaguar Gene Therapy, shares 3 important considerations for gene therapy companies. To start, the FDA is asking researchers to share data and go above the minimum requirements for their Investigational New Drug (IND) submissions.

According to, “To achieve this, innovators must develop substantial experience with their full-scale process, analytics, and compliance with Good Manufacturing Practices (GMP) prior to IND submission to fully understand the impurity profile that is the result of their process. This cannot be achieved with shaker flask experiments or running a handful of 5 or 10 liter batches. Fast and cheap is not the answer. Innovators must generate a robust body of data in order to fully understand their process capabilities and define the critical quality attributes of their important gene therapy medicines.”

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(Source: Cell and Gene, May 31^st, 2022)