White Paper

Developing Gene-Based Therapeutics

By Annmarie Kennedy, Vice President, Strategic Accounts, Oncology And Hematology; Aman Khera, Global Head Of Regulatory Strategy; and Michael F. Murphy, MD, Ph.D., Chief Medical & Scientific Officer

scientist analyzing test tube of medicine in laboratory

As of July 2020, more than 1,000 INDs have been opened in the US for gene therapy products, with 134 INDs opened in the first 7 months of 2020 alone. Researchers estimate that 30 to 60 gene therapy products will be launched for clinical usage by 2030. However, the regulatory, operational, and commercial forces that impact gene therapy development continue to evolve dramatically – not just in the US but throughout the world. For advanced therapy medicinal products (ATMPs) in general and gene therapies in particular, regulatory, operational, and commercial considerations vary from region to region, and even from country to country within a region, depending on the nature of the interventional product. There may be incomplete international harmonization of regulatory requirements – in part because requirements may remain unsettled and elusive at local and regional levels. There also may be inconsistent standards of care against which to compare the effect of an innovative therapy in terms of healthcare utilization and patient outcomes.

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