Sickle Cell Anemia affects one in 368 people of African descent, leading to painful episodes and a shorter lifespan. I first heard about this cure on Freakonomics MD, a podcast that covers the economics of medical care.
It is the first treatment using the CRISPR gene-editing system.
In the episode I listened to, they interviewed a study participant who had been fully cured of the disease using this approach to care.
Recently, the NY Times covered the progress of the treatment after a panel of experts deemed it safe for clinical use.
Read the article below. The illness afflicts more than 100,000 Americans.
Panel Says That Innovative Sickle Cell Cure Is Safe Enough for Patients
The decision by an advisory committee may lead to the Food and Drug Administration’s approval of the first treatment for humans that uses the CRISPR gene-editing system.
By Gina Kolata
Gina Kolata has reported on gene therapy for nearly 30 years and on sickle cell disease since 2018.
Oct. 31, 2023
A panel of experts said on Tuesday that a groundbreaking treatment for sickle cell disease was safe enough for clinical use, setting the stage for likely federal approval by Dec. 8 of a powerful potential cure for an illness that afflicts more than 100,000 Americans.
The Food and Drug Administration had previously found that the treatment, known as exa-cel and jointly developed by Vertex Pharmaceuticals of Boston and CRISPR Therapeutics of Switzerland, was effective. The panel’s conclusion on Tuesday about exa-cel’s safety sends it to the F.D.A. for a decision on greenlighting it for broad patient use.
Exa-cel frees patients from the debilitating and painful effects of this chronic, deadly disease. If approved, the Vertex product would be the first medicine to treat a genetic disease with the CRISPR gene-editing technique.
It could also be the first of a series of new options to cure the excruciating illness. By Dec. 20, the F.D.A. will decide on a second potential cure for sickle cell, a gene therapy devised by the company Bluebird Bio of Somerville, Mass.