One after another, the men and women testified how a new gene-editing treatment had relieved their suffering from sickle-cell disease. They addressed a Tuesday hearing in which the Food and Drug Administration heard patients and scientists weigh in on what’s likely to be the country’s first-approved genetic treatment for the disorder.
All but one of the dozens of patients treated with exa-cel in clinical trials have gone for more than a year without the blood-clogging crises of sickle cell, and have been spared the frequent hospital stays that those crises provoked. The one exception was a patient whose condition was aggravated by an infection, and his symptoms were relatively mild.
FDA documents posted online in preparation for the meeting don’t reveal great concerns at the agency over exa-cel. After reading them last week, William Blair analyst Sami Corwin wrote that they set up “a near-best-case scenario” for the meeting—and for exa-cel’s approval as a treatment for sickle cell.
At the meeting, agency reviewers were mainly concerned with the exa-cel developers’ tests for off-target DNA edits that might cause side effects. That crowded field is one reason why shares of Vertex and Crispr Therapeutics haven’t gotten much lift from exa-cel’s approaching approval. Vertex has roughly tracked the Nasdaq Composite Index, while Crispr has lagged behind them both.
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