Skip to Content
Biotechnology

Companies declare signs of success in CRISPR treatment of blood disorders

November 19, 2019
Blood cells
Blood cells
Blood cellsWikimedia

The era of editing human genes to treat inherited disease has arrived. That’s according to a pair of biotech companies that say they’ve eliminated painful symptoms from two patients with blood diseases.

The patients: One has sickle-cell disease and the other suffers from beta thalassemia. For both, the problem is too little hemoglobin, the oxygen carrier in the blood.

Earlier this year, each patient underwent an intensive treatment in which doctors collected bone marrow cells and then employed the gene-editing technology CRISPR to turn on a second copy of the hemoglobin gene, normally not active in adults. Then the cells were returned to their bodies.

The result: CRISPR Therapeutics and Vertex Pharmaceuticals, the companies leading the first-of-a-kind effort, say there are signs the treatments have worked. The beta thalassemia patient, for instance, who lives in Europe, used to receive about 16 blood transfusions a year but hasn’t needed one since the treatment. According to NPR, the sickle-cell patient, a 34-year-old named Victoria Gray, no longer suffers from the pain attacks typical of that condition.

“This preliminary data shows for the first time that gene editing has actually helped a patient with sickle-cell disease. This is definitely a huge deal,” Haydar Frangoul of the Sarah Cannon Research Institute told NPR.

Gene therapy: The apparent success of CRISPR technology expands on the growing record of positive results for gene therapy, or the idea of repairing and replacing disease-causing genes. Both patients did suffer some serious side effects, because their immune systems needed to be mostly destroyed with chemotherapy before the repaired bone marrow cells were reinfused.

High cost: Given the substantial lifelong expense of treating these blood diseases, including hospital admissions, you can expect companies to try to charge insurers prices in the millions if they get a CRISPR cure approved. For now, the biotechs don’t want to talk about the costs. Samarth Kulkarni, CEO of CRISPR Therapeutics, told Forbes it is “too early to contemplate any sort of pricing decisions.”

Deep Dive

Biotechnology

Muhammad bin Salman funds anti-aging research
Muhammad bin Salman funds anti-aging research

Saudi Arabia plans to spend $1 billion a year discovering treatments to slow aging

The oil kingdom fears that its population is aging at an accelerated rate and hopes to test drugs to reverse the problem. First up might be the diabetes drug metformin.

individual aging affects covid outcomes concept
individual aging affects covid outcomes concept

Anti-aging drugs are being tested as a way to treat covid

Drugs that rejuvenate our immune systems and make us biologically younger could help protect us from the disease’s worst effects.

Workers disinfect the street outside Shijiazhuang Railway Station
Workers disinfect the street outside Shijiazhuang Railway Station

Why China is still obsessed with disinfecting everything

Most public health bodies dealing with covid have long since moved on from the idea of surface transmission. China’s didn’t—and that helps it control the narrative about the disease’s origins and danger.

person carrying styrofoam box used for transporting human organs
person carrying styrofoam box used for transporting human organs

A new storage technique could vastly expand the number of livers available for transplant

It allows donor livers to be held for days—significantly longer than the standard now–and even treated if they are damaged.

Stay connected

Illustration by Rose WongIllustration by Rose Wong

Get the latest updates from
MIT Technology Review

Discover special offers, top stories, upcoming events, and more.

Thank you for submitting your email!

Explore more newsletters

It looks like something went wrong.

We’re having trouble saving your preferences. Try refreshing this page and updating them one more time. If you continue to get this message, reach out to us at customer-service@technologyreview.com with a list of newsletters you’d like to receive.