US FDA Nods Two Gene Therapies For Sickle Cell Disease

The U.S Food and Drug Administration (FDA) approved two gene therapies for sickle cell disease, including the first treatment in the country based on the Nobel Prize-winning CRISPR gene editing technique.

Casgevy, developed by partners Vertex Pharmaceuticals and CRISPR Therapeutics, and bluebird bio's Lyfgenia were approved for people aged 12 years and older. CRISPR, discovered by Jennifer Doudna and CRISPR Therapeutics co-founder Emmanuelle Charpentier, uses molecular "scissors" to trim faulty parts of genes that can then be disabled or replaced with new strands of normal DNA, as per economic times.

Bluebird's gene therapy works by inserting changed genes into the body via crippled viruses. Sickle cell disease is a severe, genetic blood ailment in which the body produces faulty, sickle-shaped hemoglobin, affecting red blood cells' ability to transport oxygen to the body's tissues.

The condition, which can be severe and lead to death, affects an estimated 100,000 persons in the United States, the majority of whom are African-American.

"Today's actions follow rigorous evaluations of the scientific and clinical data needed to support approval, reflecting the FDA's commitment to facilitating development of safe and effective treatments for conditions with severe impacts on human health," said Peter Marks, director of the FDA's Center for Biologics Evaluation and Research.

Sickle cells tend to clump together and can clog small blood arteries, resulting in excruciating agony. They can also cause strokes and organ failure. Both medicines helped lessen painful episodes in patients with the illness in separate clinical studies, with 29 of 31 patients receiving Casgevy and 28 of 32 patients receiving Lyfgenia showing improvement.

CRISPR Therapeutics' stock rose 1.6% in the United States, while Vertex Pharmaceuticals' stock fell 1.4%. Bluebird Bio's stock dropped 20%.