Session Details

Prenatal cell and gene therapies offer the opportunity of treating, and ideally curing, a wide range of genetic disorders. The ability to cure a genetic disorder prior to birth, or even to just preempt organ damage during development and/or prevent postnatal immunologic reactions to the missing product(s), would completely change the face of clinical care for these patients, as well as dramatically reducing the financial burden for the patient, their family, and the health care system. In this session, we will provide a brief overview of the rationale and unique advantages to treating genetic disorders prior to birth, discuss the criteria for selecting the most promising candidate diseases, and highlight some of the seminal preclinical studies that paved the way for current clinical trials, the latest results of which will also be presented. Given that the patient that will receive prenatal gene and cell therapies cannot give consent, we will also hear from leading experts who will help us to navigate the unique ethical issues surrounding the clinical application of prenatal treatments.