Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug

Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug

The new gene therapies for sickle cell disease—including the gene-editing treatment Casgevy, based on research at Boston Children’s Hospital—have been game-changing for the patients who have received them. But Stuart Orkin, MD, the Boston Children’s hematologist whose work led the way to Casgevy, wants to go even further.

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