From fiction to science: clinical potentials and regulatory considerations of gene editing

• Main Authors: Maria Schacker, Diane Seimetz
• Format: Article
• Language: English
• Published: Wiley 2019-10-01
• Series: Clinical and Translational Medicine
• Subjects: Gene editing; Gene therapy; Drug development; Regulatory strategy; Clinical trials; Artificial intelligence
• Online Access: http://link.springer.com/article/10.1186/s40169-019-0244-7

Abstract Gene editing technologies such as CRISPR/Cas9 have emerged as an attractive tool not only for scientific research but also for the development of medicinal products. Their ability to induce precise double strand breaks into DNA enables targeted modifications of the genome including selective knockout of genes, correction of mutations or precise insertion of new genetic material into specific loci. Gene editing-based therapies hold a great potential for the treatment of numerous diseases and the first products are already being tested in clinical trials. The treatment indications include oncological malignancies, HIV, diseases of the hematopoietic system and metabolic disorders. This article reviews ongoing preclinical and clinical studies and discusses how gene editing technologies are altering the gene therapy landscape. In addition, it focusses on the regulatory challenges associated with such therapies and how they can be tackled during the drug development process.