Food and Drug Administration Guidance on Design of Clinical Trials for Gene Therapy Products with Potential for Genome Integration or Genome Editing and Associated Long-Term Follow-Up of Research Subjects.

Abstract

Introduction: With the burgeoning growth of the gene therapy industry, the Food and Drug Administration (FDA) has produced various guidance documents intended to help gene therapy manufacturers design their preclinical testing and clinical trials to facilitate the process of obtaining marketing approval.

Discussion: Biosafety professionals and institutional biosafety committees (IBCs) with oversight of clinical trials or biopharmaceutical manufacturing stand to benefit from understanding how these guidance documents set the standard for writing the clinical research protocols that are reviewed by IBCs. Although the FDA guidance documents are typically meant for manufacturers (either pharmaceutical companies serving as research sponsors or investigators at academic institutions), much of the content is useful for biosafety professionals and IBCs during the IBC review process.

Conclusion: This article specifically addresses guidance documents pertaining to gene therapy vectors capable of genomic integration, testing for replication competent retrovirus, genome editing, and long-term follow-up of research subjects.