Skip to Content
Biotechnology and health

The first gene-editing treatment: 10 Breakthrough Technologies 2024

Sickle-cell disease is the first illness to be beaten by CRISPR, but the new treatment comes with an expected price tag of $2 to $3 million.

a scientist looks at a tall strand of DNA in a suspension of liquid. A hose sends the liquid back to an IV and into the arm of a patient seated comfortably in a domestic chair with two nice plants and a happy, observant cat.
Simon Landrein

WHO

CRISPR Therapeutics, Editas Medicine, Precision BioSciences, Vertex Pharmaceuticals

WHEN

Now

The first gene-editing cure has arrived. Grateful patients are calling it “life changing.”

It was only 11 years ago that scientists first developed the potent DNA-snipping technology called CRISPR. Now they’ve brought CRISPR out of the lab and into real medicine with a treatment that cures the symptoms of sickle-cell disease.

Sickle-cell is caused by inheriting two bad copies of one of the genes that make hemoglobin. Symptoms include bouts of intense pain, and life expectancy with the disease is just 53 years. It affects 1 in 4,000 people in the US, nearly all of them African-American. 

So how did this disease become CRISPR’s first success? A fortuitous fact of biology is part of the answer. Our bodies harbor another way to make hemoglobin that turns off when we’re born. Researchers found that a simple DNA edit to cells from the bone marrow could turn it back on.

Many CRISPR treatments are in trials, but in 2022, Vertex Pharmaceuticals, based in Boston, was first to bring one to regulators for approval. That treatment was for sickle-cell. After their bone marrow was edited, nearly all the patients who volunteered in the trial were pain free. 

Good news. But the expected price tag of the gene-editing treatment is $2 to $3 million. And Vertex has no immediate plans to offer it in Africa—where sickle-cell disease is most common, and where it still kills children.

The company says this is because the treatment regimen is so complex. It involves a hospital stay; doctors remove the bone marrow, edit the cells, and then transplant them back. In countries that still struggle to cover basic health needs, the procedure remains too demanding. So simpler, cheaper ways to deliver CRISPR could come next. 

Deep Dive

Biotechnology and health

This baby with a head camera helped teach an AI how kids learn language

A neural network trained on the experiences of a single young child managed to learn one of the core components of language: how to match words to the objects they represent.

The next generation of mRNA vaccines is on its way

Adding a photocopier gene to mRNA vaccines could make them last longer and curb side effects.

An AI-driven “factory of drugs” claims to have hit a big milestone

Insilico is part of a wave of companies betting on AI as the "next amazing revolution" in biology

Ready, set, grow: These are the biotech plants you can buy now

For $73, I bought genetically modified tomato seeds and a glowing petunia.

Stay connected

Illustration 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.