Hemoglobin– tag –
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CRISPR Gene Therapies Expand in 2026: EDIT-101 for Retinitis Pigmentosa Gains FDA Accelerated Approval, Following Casgevy for Sickle Cell Disease
Ubie Doctor's Note Japan Overview By 2026, CRISPR-based gene therapies are rapidly expanding, with EDIT-101 for retinitis pigmentosa receiving FDA accelerated approval in late 2025, building on the 2023 approval of Casgevy for sickle cel... -
Vertex’s CRISPR Gene Therapy CASGEVY Gains Expanded FDA Approval for Sickle Cell Disease and Beta Thalassemia in Children Ages 2 and Older
Vertex Pharmaceuticals USA Overview Vertex Pharmaceuticals announced the US FDA has approved an expanded indication for its gene therapy, CASGEVY® (exagamglogene autotemcel), for treating sickle cell disease or transfusion-dependent beta... -
HCA Healthcare Announces Promising Results for CRISPR Gene Therapy Exa-cel in Pediatric Sickle Cell Disease and Beta-Thalassemia Patients
HCA Healthcare USA Overview HCA Healthcare has announced findings from a New England Journal of Medicine study, sponsored by Vertex Pharmaceuticals, demonstrating that the CRISPR gene-editing therapy exa-cel shows promising results in pe... -
Vertex Pharmaceuticals Secures CASGEVY Reimbursement Agreement in Germany for Sickle Cell Disease and Beta Thalassemia
Vertex Pharmaceuticals USA Overview Vertex Pharmaceuticals announced a reimbursement agreement in Germany for CASGEVY (exa-cel), a groundbreaking gene-editing therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassem...
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