FDA Approves Gene Therapies for Sickle Cell Disease

Two cell-based gene therapies will address the debilitating hereditary blood disorder.

The U.S. Food and Drug Administration (FDA) has granted approval of two groundbreaking cell-based gene therapies, Casgevy and Lyfgenia, marking a pivotal development in the treatment of sickle cell disease for patients aged 12 and above. The revolutionary genome editing technology treats the hereditary blood disorder, which affects approximately 100,000 individuals in the U.S., particularly minority populations. A mutation in hemoglobin causes red blood cells to adopt a sickle shape, impeding blood flow and oxygen delivery, which leads to severe pain and organ damage.

“Sickle cell disease is a rare, debilitating, and life-threatening blood disorder with significant unmet need, and we are excited to advance the field, especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Office of Therapeutic Products Director Nicole Verdun in the FDA’s Center for Biologics Evaluation and Research.

As the Lord Leads, Pray with Us…

  • For U.S. scientists and researchers as they seek therapies and treatments for diseases and disorders.
  • For FDA Commissioner Califf and officials in the agency as they review medications and treatment proposals.
  • For healing for those who suffer from sickle cell disease.

Sources: Food and Drug Administration


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