Developing Gene-Based Therapeutics: Strategic Considerations For Regulatory, Operational, And Commercial Success

By Aman Khera, Global Head of Regulatory Strategy; and Michael F. Murphy, MD, Ph.D., Chief Medical & Scientific Officer

As of July 2020, over 1,000 Investigational New Drug (IND) applications have been filed in the United States for gene therapy products, with 134 of these applications submitted in just the first seven months of that year. Researchers anticipate that between 30 and 60 gene therapy products will be introduced for clinical use by 2030. However, the landscape of gene therapy development is undergoing significant changes, influenced by a range of evolving regulatory, operational, and commercial factors, both in the United States and globally.

The development of advanced therapy medicinal products (ATMPs), including gene therapies, is subject to a complex web of regulations and operational practices that vary widely across different regions and countries. This variation is partly due to the incomplete international harmonization of regulatory standards, as requirements can remain unsettled or unclear at local and regional levels. Additionally, there may be inconsistencies in the standards of care used to evaluate the effectiveness of innovative therapies, which can impact healthcare utilization and patient outcomes. As a result, the path to clinical application and market approval for gene therapies is often complex and influenced by a myriad of factors specific to each region and country.