White Paper

Reducing The Risk During Cell And Gene Therapy Development And Manufacturing

By Martin Wisher, Manjula Aysola, and Natika Calhoun


Recently approved cell and gene therapies are delivering impressive results for patients who otherwise have exhausted all treatment options or have had no options available to them. The combination of these results, along with a deeper understanding of the underlying biology, is fueling explosive growth within the industry as evidenced by robust pipelines.

While these new modalities are generating significant excitement among patients and caregivers, cell and gene therapy manufacturers are experiencing growing pains. The rapid growth and continued momentum of the industry has led to the issuance of new guidance. The US FDA finalized the guidance for several aspects of development of gene therapies in January 2020. This previous lack of clarification and the current novelty of the guidance contributes to the complexity and risk of advancing these novel therapeutics. Success in the complex and highly competitive cell and gene therapy space requires managing uncertainties and risks related to manufacturing. This imperative can prove especially challenging when operating with compressed regulatory timelines and a sense of urgency to address significant unmet patient needs.

In this article we share insights for navigating within a novel and complex regulatory environment, mitigating risks posed by raw materials and implementing proper risk assessments during manufacturing. We also share our perspective on optimizing communication and ensuring transparency when working with a contract development and manufacturing organization (CDMO).

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