Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate

The promise of gene therapy looms large for families dealing with rare, genetic disorders. Such treatments offer the possibility of one-time cures. But families and researchers worry such therapies will remain out of reach. About 350 million people worldwide suffer from rare diseases, most of which are genetic. But the individual disorders affect relatively few people. So there’s little commercial incentive to develop or bring these treatments to market. Scientists say this dynamic threatens to curb research and progress across the nascent field. Scientists, patients and families are scrambling for solutions, often turning to charitable organizations, patient groups and governments.