Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing
The FDA has released a draft guidance document concerning the use of prior knowledge in the development of human gene therapy products that incorporate genome editing technologies.
Manufacturers and developers of human gene therapy products utilizing genome editing, as well as regulatory and clinical development departments managing these portfolios.
The release of this draft guidance suggests the FDA is establishing a framework for how developers can use existing data to streamline or support the development of genome-edited products. This could potentially reduce the burden of de novo data generation for subsequent products in a platform, provided the 'prior knowledge' is scientifically justified. Regulatory professionals should note this as an indication of the agency's evolving thinking on platform-based development for complex biologics.
Regulatory teams should evaluate existing internal and external data sets to determine how prior knowledge can support future IND or BLA submissions. QA and R&D should ensure that data used for 'leveraging' is sufficiently robust and relevant to the specific genome editing platform under development.
This is the Draft Guidance for Industry, Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing
Open in openFDA / FDA.gov