Genetic Therapies for Rare Diseases: Developing Ethical Regulatory Policies

Last year, researchers saved an infant named KJ from a life-threatening rare metabolic disorder using a customized gene editing therapy. This was the first time that an individualized gene therapy was used to treat a human patient, and it has stirred hope. The next frontier is scalability.

There are over 10,000 rare diseases, 80% of which have a genetic cause, making them particularly amenable to yet-to-be-developed gene-based therapies. In recent years, dozens of precision genetic medicines have been approved, many for the treatment of rare diseases. Still, approximately 95% of rare diseases lack an approved drug. How can we transition from dozens of approvals to hundreds or even thousands, so that more rare disease patients and families have effective treatments? 

To boost innovation in gene-based therapies, it is important to address not only scientific, manufacturing, and commercial challenges, but also ethical and regulatory issues. Bioethicists have an obligation to advocate for regulatory policies that support the development of safe and effective genetic therapies for rare diseases and enable...