The US Food and Drug Administration (FDA) has finalized guidance to assist sponsors developing gene therapy products that incorporate genome editing (GE) of human somatic cells; the final version revises the draft by stating that the accelerated approval pathway may be used for these products.
The final guidance, issued on 29 January, addresses the information that should be provided in an investigational new drug (IND) application to assess the safety and quality of investigational GE products, and covers product manufacturing and testing, nonclinical safety assessment, and clinical trial design. It also provides more clarity on the criteria for enrolling pediatric patients in clinical trials and provides clearer expectations for potency testing…
