5 Key Takeaways

• 1

  Manufacturing or quality deficiencies accounted for 74% of Complete Response Letters issued by the FDA from 2020 to 2024.

• 2

  Three high-profile cell and gene therapy programs faced delays or rejections in 2025 due to Chemistry, Manufacturing, and Control-related issues.

• 3

  Early development decisions in gene therapy have significant downstream consequences, as emphasized by FDA guidance on CMC.

• 4

  A cohesive QC strategy is essential, as it cannot be redesigned at each development stage without risking compliance and efficiency.

• 5

  Geographical considerations in quality strategy are crucial, as operational habits can lead to documentation inconsistencies across regions.