U.S. poised to approve breakthrough treatment

At age 19, Joe Tsogbe underwent his first hip replacement. In his 20s, he averaged about nine hospitalizations a year. By his 30s, that rose to more than a dozen. 

All the result of sickle cell disease, an inherited blood disorder where a genetic mutation causes normally full-moon shaped red blood cells to form into half moons and get stuck inside blood vessels, restricting blood flow and causing bouts of excruciating pain. 

The disease affects about 100,000 people in the U.S., many of whom are Black. Few treatments are available, and the only cure is a bone marrow transplant where a patient receives healthy blood stem cells from a donor. New genetic treatments aim to offer relief while eliminating the need to track down donors.

Tsogbe, now 37, received one of those options, known as exa-cel and co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics, via a clinical trial in 2021. The treatment uses Nobel Prize-winning technology called CRISPR to edit a person’s DNA and alleviate the symptoms of sickle cell disease. 

U.S. regulators are expected to approve exa-cel for use in sickle cell patients by the end of this week. The U.K. approved it under the brand name Casgevy last month.

Regulators in the U.S. are also reviewing another gene therapy from Bluebird Bio called lovo-cel. It works differently than exa-cel but is administered similarly and is also intended to eliminate pain crises. It’s expected to be approved later this month.

Approval of exa-cel by the U.S. Food and Drug Administration would mark a scientific milestone about a decade after the discovery of CRISPR and a breakthrough for patients desperate for a better option.

It could also present a major test for the American health-care system, with Wall Street eyeing a price tag of around $2 million per patient. Tens of thousands of people could be eligible. 

In 2012, researchers Jennifer Doudna and Emmanuelle Charpentier published their seminal paper on a system for…