Supporting the need for global regulatory harmonization

The recent stabilization of baby KJ Muldoon’s condition following CRISPR-based treatment for CPS1 deficiency exemplifies the real-world impact of advanced therapy medicinal products (ATMP) innovation. The case underscores the importance of coordinated efforts among researchers, clinicians, regulators and manufacturers to accelerate the development and delivery of ATMPs. However, while ATMP scientific breakthroughs are advancing rapidly, progress is constrained by regulatory complexity, financial risk and market access barriers. In response, we brought together more than 30 experts from 20 companies, demonstrating leadership by aligning on best practices and proposing practical solutions to regulatory barriers. These collaborative efforts are documented in our new paper, which explores subjects such as regulatory challenges in ATMP development, market and investment barriers, and regulatory engagement and expedited pathways. To address the challenges, the workstream proposes several regulatory changes. These include: • adopting flexible clinical trial designs • endpoints tailored to the unique nature of ATMPs • a two-tier regulatory system (like China’s) to allow the faster initiation of first-in-human trials under local ethics review boards. Our paper showcases BioPhorum ATMP’s pivotal role in helping the wider industry conversation to move from a fragmented regulatory experience to aligned, evidence-based action. It brings real-world challenges into a clear industry viewpoint, demonstrating where regulatory misalignment slows development and patient access. Instead of advocating for deregulation, we are reframing the discussion around regulatory evolution, championing harmonization, flexibility and clarity while prioritizing patient safety. This approach elevates industry dialogue by turning isolated regulatory challenges into shared themes that regulators and policymakers can address collaboratively.