Key questions (and answers) about the historic approval of a CRISPR-based medicine

History just happened.

For the first time, a regulator has cleared a treatment using CRISPR, the gene-editing technology, for patients. The regulator is the United Kingdom’s Medicines and Healthcare products Regulatory Agency. The product is Casgevy, a treatment for sickle cell disease and beta thalassemia, two blood disorders. It was developed by CRISPR Therapeutics, the Swiss company co-founded by Nobel laureate Emmanuelle Charpentier, and Vertex Pharmaceuticals, a large Boston-based biotech firm.

In 2012, when Charpentier and Jennifer Doudna published their landmark paper on editing DNA in a test tube with CRISPR, almost no one believed that a treatment based on the technology would be reaching patients in little more than a decade.

The news is perhaps even bigger because Casgevy is directed at sickle cell disease, a painful and life-shortening disorder that causes red blood cells to become misshapen. It afflicts 100,000 people in the U.S., and is far more common in people of African descent. It was long ignored by researchers and drug companies, although that is changing.

But history is also complicated. This first CRISPR treatment is not an easy treatment to receive, requiring patients to spend weeks, even months, in the hospital before and after the therapy is administered.

The U.S. Food and Drug Administration is expected to approve Casgevy on or before Dec. 8. CRISPR technology’s ease of use, efficiency, and customizability make it an ideal way to treat disorders caused by genetic mutations, and Casgevy is likely to be just the first of many treatments for inherited diseases…

“I don’t think we want to live, or I don’t want to live, in a world where only a few wealthy or connected people can get access to something like this,” Jennifer Doudna told STAT. She said that researchers at the Innovative Genomics Institute, which she runs, are focused “on this question of how you make sure that CRISPR is going to ultimately be available to people worldwide who can benefit from it.”

“It’s a big challenge,” Doudna said.







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