FDA Considers Safe and Promising CRISPR Gene Editing Treatment for Sickle Cell Disease
The US Food and Drug Administration (FDA) is considering the approval of exa-cel, a groundbreaking gene editing treatment for sickle cell disease. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, exa-cel would be the first FDA-approved treatment that uses CRISPR technology. The treatment involves altering the patient's own stem cells with CRISPR to correct the genetic problems causing sickle cell, and then reintroducing the modified stem cells to the patient. In company studies, the treatment showed positive results, with a significant reduction in painful crises experienced by patients. The FDA sought advice from an independent panel to evaluate the treatment's safety and determine the criteria for approval. The FDA is expected to make a decision by December 8.
- FDA considers first CRISPR gene editing treatment that may cure sickle cell CNN
- Panel Says That Innovative Sickle Cell Cure Is Safe Enough for Patients The New York Times
- FDA considers gene therapy for sickle cell disease: Cure at a cost. USA TODAY
- CRISPR, Vertex gene editing therapy gets warm response from FDA panel Seeking Alpha
Reading Insights
0
1
6 min
vs 7 min read
90%
1,226 → 117 words
Want the full story? Read the original article
Read on CNN