Dec 19, 2017

FDA Just Approved The First-Ever Gene Therapy For an Inherited Disease

Posted by in categories: biotech/medical, genetics

In a historic move, the Food and Drug Administration on Tuesday approved a pioneering gene therapy for a rare form of childhood blindness, the first such treatment cleared in the United States for an inherited disease.

The approval signals a new era for gene therapy, a field that struggled for decades to overcome devastating setbacks but now is pushing forward in an effort to develop treatments for haemophilia, sickle-cell anaemia, and an array of other genetic diseases.

Yet the products, should they reach patients, are likely to carry stratospheric prices – a prospect already worrying consumer advocates and economists.

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