Navigating The Regulatory Landscape Of Cell And Gene Therapies

The burgeoning field of cell and gene therapy is marked by rapid advancements, yet its commercialization hinges on navigating a complex regulatory maze. This blog, a follow-up to our 2024 ThinkLive Cell and Gene Therapy Summit, delves into the critical regulatory considerations, challenges, and best practices for companies operating in this space.

From defining therapeutic editing thresholds to streamlining interactions with multiple regulatory agencies, the FDA and MHRA have issued comprehensive guidance. However, the complexities of these regulations necessitate a deep understanding of specific modalities, a flexible supply chain, and early consideration of potency assays and data collection.

While advancements like whole genome and RNA sequencing are crucial for characterizing cell and gene therapy products, regulatory incentives like orphan drug designation are increasingly utilized to address the unique challenges of developing therapies for small patient populations.

Despite progress made by regulatory agencies, differences persist in facility requirements and GMP standards, particularly in Europe. Companies must actively educate regulators about advancements and challenges in the field, while engaging with payers and HTA bodies to ensure successful development and commercialization.

Learn more by reading the full article.