Nov 20, 2019

CRISPR-based gene editing treatments benefit patients with blood disorders

A researcher observes a CRISPR/Cas9 process through a stereomicroscope
A researcher observes a CRISPR/Cas9 process through a stereomicroscope. Photo: Gregor Fischer/picture alliance via Getty Images

The first two patients to receive CRISPR-based treatments for their blood disorders benefited from the therapy, the two companies developing the treatment announced yesterday — a sign that gene editing may be a safe and effective way to cure blood diseases, STAT reports.

Why it matters: The two blood disorders, sickle cell disease and beta thalassemia, can have a drastic impact on patients' lives. A cure would be transformative.

Details: The two patients have been free from blood transfusions and disease symptoms for a relatively short period of time and have experienced only short-term, treatable side effects.

The big picture: CRISPR Therapeutics and Vertex Pharmaceuticals, the companies developing the treatment, aren't the only ones actively looking for new treatments for these blood disorders.

  • Bluebird Bio's gene therapy for beta thalassemia was approved in Europe in June, and it's expected to be approved in the U.S. next year. Other companies and academics are also looking for gene editing or gene therapy treatments for these diseases.

"For decades, we knew about the sickle cell disease mutation but we didn’t know about other genes [involved in the disease] and we didn’t have the necessary tools for genetic correction” of cells, Mitchell Weiss of St. Jude Children’s Research Hospital told STAT.

  • "Now we have a confluence of scientific understanding and technology that can come together to make things happen."

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Editor's note: This piece has been corrected to show that Bluebird Bio's gene therapy for beta thalassemia was approved in Europe this year (not last year).

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