Gene therapies have the potential to offer a literal lifeline to people with rare diseases, many of which have no effective treatments. While each rare disease only affects a small number of patients, scientists have identified about 7,000 of them.
But despite considerable progress in gene therapy research over the past few decades, only two rare disease treatments — one for an inherited form of blindness, another for the neuromuscular disorder spinal muscular atrophy — have been approved for use in the U.S. A number of recent safety and manufacturing setbacks have also stymied progress, concerns highlighted at an FDA meeting in September.
