Please provide your email address to receive an email when new articles are posted on . Exagamglogene autotemcel helped patients with beta-thalassemia achieve transfusion independence. The drug also ...
Industry Focus eBook - Cell & Gene Therapy (1st Edition) eBook Industry Focus: Cell & Gene Therapy Download the latest edition At 48 months past infusion, adults had a mean score improvement of 14.0 ...
As Vertex Therapeutics nears completion of a historic FDA submission, the rare disease specialist has depicted a rosy launch picture for what could become the first CRISPR-based gene editing therapy.
Data from the study showed that exa-cel prevented vaso-occlusive crises in all but 1 of the 30 evaluable patients for at least 12 months. Exagamglogene autotemcel (exa-cel; Casgevy) prevented ...
At age 45, Dr. Lakiea Bailey said, for the longest time, that she was the oldest person with sickle cell anemia that she knew. The executive director of the nonprofit patient advocacy group the Sickle ...
(ORLANDO, Dec. 6, 2025) Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an effective cure for beta-thalassemia and sickle cell ...
A group of independent experts to the US FDA for the most part embraced a first of its kind gene editing therapy developed by CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharma (NASDAQ:VRTX) for ...
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