FDA Provides Guidance on Human Genome Editing in Gene Therapies
The ultimate guidance offers suggestions for creating gene therapy products that involve editing the genome of human somatic cells.
The FDA released a comprehensive guidance document on Jan. 30, 2024, regarding Human Gene Therapy Products Incorporating Human Genome Editing. The document offers suggestions for creating gene therapy products that involve genome editing of human somatic cells, with a focus on simplicity in teaching. Here are some suggestions for including the necessary information in an investigational new drug application to evaluate the safety and quality of GE products. This information covers various aspects including product design, manufacturing and testing, nonclinical safety assessment, and clinical trial design.
As per the agency's report, there has been a significant rise in the utilization of human GE, leading to the swift advancement of gene therapy products that integrate GE. The FDA cautions that there is limited knowledge about the potential risks associated with these products. The guidance document provides methods for evaluating the safety of these products. The agency mentions in the document that it may review its suggestions as the field progresses.
The guidance document provides a clear definition of human genetic engineering. It explains that this process involves making changes to the DNA sequences in human somatic cells, either outside or inside the body, using various genetic engineering technologies. This guidance refers to human gene therapy products that incorporate genetic engineering as human GE products. The evaluation process for human GE products conducted by the FDA is based on scientific principles, carefully considering the advantages and potential drawbacks of each product. The profile of benefits and risks for each product varies based on the proposed indication and patient population, as well as the extent and duration of therapeutic benefit achieved, and the availability of alternative therapeutic options. Certain risks come with using GE approaches, such as off-target editing, unintended consequences of on-target editing, and the uncertainty surrounding the long-term effects of both on- and off-target editing.
The document covers various aspects such as GE methods, types and levels of genomic modification, and methods of delivering GE components. Chemistry, manufacturing, and controls recommendations are also covered.
Source: FDA
