Cell and gene therapies offer great promise for rare diseases, where patients—many of them children—may be severely ill or debilitated and treatment options are often limited. Development of these advanced therapeutics is complex due to both disease-specific and treatment-related factors and executing clinical trials that meet the expectations of regulators requires careful attention to study design and patient safety. In this guide, we review the current landscape of cell and gene therapy (CGT) and provide guidance on key challenges to overcome and questions to address when designing trials in rare diseases.
