The Impact of CRISPR 3.0 in Healthcare: Its Promise and Challenges Ahead

Monday - December 25 2023, 10:46 UTC - 11 months ago

This article reflects on the biotechnology coverage over the past year, and specifically focuses on the gene editing technique, CRISPR 3.0. The article outlines its promise, its cost, and its challenges. It notes that the FDA have not approved its use in plants, and discusses ways to reduce the costs associated with it.

Welcome back to The Checkup. This will be our last issue of 2023, so this week I've been reflecting on our biotechnology coverage over the past year. As I scrolled through our archives, I was struck by the vast number of stories we wrote about gene editing.

CRISPR 3.0, which allows scientists to replace bits of DNA or insert new chunks of genetic code, is still being tested in animals. One company, Prime Medicine, plans to seek FDA approval to launch a human trial of a treatment for chronic granulomatous disease, a genetic immune disorder, in 2024.

Given the complexity of the treatment, you won't be surprised to learn that it's expensive—it costs an estimated $2.2 million. That price tag means it's out of reach for many, especially people in low-income countries.

According to Arnold Altshuler, who leads business development at Prime Medicine, the key to getting the treatment to those who need it is developing a more cost-effective delivery system. "One question I get a lot is: How are we going to get to the rest of the world?" Altshuler said. "And I think the answer is not by trying to do bone-marrow transplants in the rest of the world. It's just too resource intensive, and the infrastructure is not there. I think the goal will be achieved sooner by finding another modality, like a pill that can be distributed much more effectively." .

It's possible, though, that CRISPR 3.0 may not be the only tool available for treating genetic conditions. Gene editing is just one part of the larger field of biotechnology, which is also researching methods such as stem cell therapies, gene therapies, and pharmacological treatments.

Moreover, scientists are looking into ways to reduce the costs associated with using CRISPR 3.0. For example, they are improving the microbes and other organisms used to transfer DNA fragments and developing methods of editing genes with minimum harm to the patient.

The FDA has not yet approved CRISPR 3.0 for use in plants, but this may change in the future. For now, the focus is on getting the technique approved for use in human trials, and this could pave the way for much-needed treatments for rare and inherited diseases that are not covered by existing treatments.