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The regulatory landscape for cell and gene therapies (CGTs) is shifting fast. From increased scrutiny on Chemistry, Manufacturing, and Controls (CMC) to operational challenges at the U.S. Food and Drug Administration (FDA), sponsors must be prepared to adapt with foresight and flexibility.
Recent FDA staffing reductions and resource constraints have raised concerns across the industry. While core regulatory reviews remain largely on schedule, the agency is under pressure, and sponsors must be more proactive than ever in their planning and engagement.
👉 Why Cell and Gene Therapies Require a New Development Mindset
Unlike small molecules, cell and gene therapies are biologically complex, often personalized, and sensitive to even the smallest process changes. In this space, the phrase “the process is the product” takes on real meaning.
As Lisa Erickson, Associate Director, Regulatory Affairs at Veristat, explains, “In cell and gene therapy, early-phase meetings with FDA can’t be theoretical. Sponsors need to arrive with clear plans, data to support their proposals, and a strong understanding of both risk and readiness.”
CGT sponsors must place equal weight on CMC strategy as they do on clinical development. That means building a high-level roadmap—and a detailed, tactical plan—that identifies critical control points, potential risks, and manufacturing contingencies well before issues arise.
Hear from Lisa as she shares her insights on how sponsors can navigate regulatory uncertainty, build resilient CMC strategies, and stay on track in the fast-moving world of cell and gene therapy.
👉 Managing Complexity Through Comparability
Manufacturing changes are often inevitable in CGT development, whether due to scale-up, site changes, or process optimization. Each change brings the challenge of comparability: proving that pre- and post-change material is sufficiently similar in quality, safety, and efficacy.
Comparability planning should begin early, well before such changes are implemented. This involves mapping analytical and clinical needs by phase and incorporating real-time and stress condition stability data to support any changes that may arise.
Drawing from her experience, Lisa notes that failure to plan appropriately can lead to costly consequences—additional clinical studies, delayed filings, or even restarting development.
👉 Strategic Engagement Is More Important Than Ever
Given current FDA resource constraints, sponsors must be thoughtful in their communication. “It’s not just about submitting questions—it’s about submitting the right questions, backed by the right data,” Lisa says. High-quality regulatory engagement improves outcomes for both sponsors and reviewers, especially in today’s environment.
At Veristat, we support clients with both the strategic foresight and hands-on expertise to navigate evolving regulatory challenges. Our embedded teams help develop robust CMC plans that align with FDA expectations and anticipate the realities of complex manufacturing landscapes.
👉 Ready to Learn More?
Maintaining strong relationships and interactions with key global regulatory agencies is critical. Our team has deep expertise in all accelerated pathways to approval, and in applying for and retaining special designations. In the past 5 years, Veristat has completed more than 20+ cell and gene therapy clinical trial applications and marketing applications. Through ongoing regulatory engagement, we keep up with the constantly changing landscape for advanced cell and gene therapy products.
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