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What is gene therapy and how does it work?




The Food and Drug Administration on Thursday approved a treatment that uses gene therapy to treat severe hemophilia A, a rare and sometimes fatal blood disorder.

The new drug, Roctavian, can save people with the severe form of the disease from a life of frequent injections. The drug̵[ads1]7;s maker expects that about 2,500 of the estimated 6,500 Americans with severe hemophilia A will be eligible to receive the drug with its initial approval.

The treatment uses a single infusion to insert the genes people with hemophilia A lack, and can work for years, according to data presented to regulators. It’s also the latest FDA-approved gene therapy, heralding what some researchers say is the future of treating thousands of diseases.

The FDA has approved gene therapy for conditions including sickle cell anemia and spinal muscular atrophy. Use of the technology is relatively rare, partly because gene therapy is among the most expensive drugs in the world. Roctavian will cost $2.9 million for a single infusion; Hemgenix, which treats another form of hemophilia, costs $3.5 million per use, according to Reuters. Still, researchers say the technology will be a crucial part of 21st century medicine.

Here’s what you need to know.



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