In this guidance, we, FDA, are providing recommendations to sponsors developing human genetherapy products incorporating genome editing (GE) of human somatic cells. This guidance provides recommendations regarding information that should be provided in an Investigational New Drug (IND) application in order to assess the safety and quality of the investigational GE product.
In this guidance, we, FDA, are providing recommendations to sponsors developing human genetherapy products incorporating genome editing (GE) of human somatic cells. Specifically,this guidance provides recommendations regarding information that should be provided in an Investigational New Drug (IND) application in order to assess the safety and quality of the investigational GE product, as required in Title 21 of the Code of Federal Regulations 312.23 (21 CFR 312.23). This includes information on product design, product manufacturing and testing, nonclinical safety assessment, and clinical trial design.
In general, FDA’s guidance documents, including this guidance, do not establish legally enforceable responsibilities. Instead, guidances describe the Agency’s current thinking on a topic and should be viewed only as recommendations, unless specific regulatory or statutory requirements are cited. The use of the word should in Agency guidance means that something is suggested or recommended, but not required.
Source: FDA
Docket Number:FDA-2021-D-0398
Issued by:Center for Biologics Evaluation and Research
Download Link:https://www.fda.gov/media/156894/download