Nov 20, 2019

CRISPR-based gene editing treatments benefit patients with blood disorders

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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FDA approves drug with annual cost of $442,000 for rare genetic disorder

Photo: Al Drago/CQ Roll Call

The FDA has approved the drug Givlaari, made by Alnylam Pharmaceuticals, for adults who have acute hepatic porphyria, a genetic disorder that leads to severe abdominal pain and affects just 1 in 25,000 people.

The big picture: Alnylam set Givlaari's net price, after discounts to insurers, at $442,000 per year, or about $39,000 per vial. It's a lofty price tag, but those prices are common among drugs that use new scientific techniques to treat very rare diseases.

Go deeperArrowNov 21, 2019

China is racing into CAR-T development

A lab technician prepares to thawing blood bag before genetically modifying a patients immune cells. Photo: Gerard Julien/AFP/Getty Images

China's aggressive focus on prescription drugs includes developing its own version of CAR-T, one of the most ambitious therapies on the market, Bloomberg reports.

Why it matters: Chinese scientists are attempting to develop CAR-T therapies — which genetically engineer a patient's own immune cells to destroy cancer cells — much faster and with a much cheaper price tag than those in the U.S.

Go deeperArrowDec 11, 2019

Report: Huge oversights in Chinese scientist's gene-edited babies attempt

Illustration: Sarah Grillo/Axios Visuals

MIT Technology Review released excerpts of Chinese scientist He Jiankui's unpublished research on Tuesday, underscoring massive ethical and practical oversights in his claim that he successfully used the gene-editing tool CRISPR to create genetically engineered children.

The bottom line: A primary goal of the experiment was to test if cells could be gene-edited to become HIV resistant. This could have been done without creating human test subjects, MIT notes.

Go deeperArrowDec 4, 2019