CRISPR Gene-Editing Drug Shows Promise for Sickle Cell and Thalassemia Treatment
TL;DR
The FDA has indicated that it could approve Crispr Therapeutics' gene-edited sickle cell treatment, exa-cel, as early as December. The drug works by increasing hemoglobin levels in patients with sickle cell disease and beta thalassemia. If approved, exa-cel would be the first drug using CRISPR-based gene editing to hit the market. The companies are also working to file for approval in Europe and the U.K.
- CRSP Stock Pops As FDA Sets December Data To Potentially OK Gene-Editing Drug | Investor's Business Daily Investor's Business Daily
- Vertex-CRISPR drug posts phase 3 wins, secures PDUFA date FierceBiotech
- FDA Accepts Biologics License Applications for exagamglogene autotemcel (exa-cel) for Severe Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia Yahoo Finance
- CRISPR therapy offers lasting benefits for sickle cell, thalassemia STAT
- Editas posts early sickle cell data as it races to catch Vertex FierceBiotech
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