The Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research regulates an array of diverse and complex biological products, both investigational and licensed, including human cell and gene therapy products and human cells and tissues. FDA’s oversight includes ensuring that human cells and tissues intended for transfer into a human recipient, including reproductive cells and tissues, are free from infectious diseases.
Mitochondria contain a small amount of genetic material (DNA) that is separate from the DNA in the cell’s nucleus. The mitochondrial DNA is passed down only from mother to child, and thus is inherited differently from the nuclear DNA. Mitochondrial Replacement Technology (MRT) using donor mitochondria represents a possible treatment for mitochondrial disorders, but introduces a genetic modification and raises safety concerns.
The clinical use of MRT in the United States falls within FDA’s regulatory authority. Since December 2015, Congress has included provisions in annual federal appropriations laws that prohibit FDA from accepting applications for clinical research using MRT. Therefore, clinical research using MRT in humans cannot legally proceed in the United States. FDA maintains the authority to investigate and take enforcement action in the event that it becomes aware of noncompliance with the laws and regulations administered by FDA.