Business Wire

Positive Results From Pivotal Trials of exa-cel for Transfusion-Dependent Beta Thalassemia and Severe Sickle Cell Disease Presented at the 2023 Annual European Hematology ...

BOSTON & ZUG, Switzerland--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) today announced that both pivotal trials for exagamglogene ...
The American Journal of Managed Care

Phase 3 Data Show Exa-Cel Prevents Vaso-Occlusive Crises for At Least 1 Year in SCD

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 ...
Seeking Alpha

CRISPR Therapeutics: Positive Exa-Cel AdCom Opens Door For Historic Approval

CRISPR Therapeutics AG and Vertex Pharmaceuticals are one step closer to gaining approval for their CRISPR/Cas9 gene editing therapy for Sickle Cell Disease and beta thalassemia. An FDA Advisory ...
The American Journal of Managed Care

Nearly All Patients Who Received Exa-Cel for SCD Are Hospital Free After a Year, Data Show

Haydar Frangoul, MD, MS, medical director, Pediatric Hematology/Oncology for Sarah Cannon Research Institute at TriStar Centennial Children’s Hospital, Nashville, presented the data on the long-term ...
FiercePharma

Vertex, CRISPR score landmark FDA approval for sickle cell disease gene therapy Casgevy

Groundbreaking? Game-changing? Transformational? Historic? None of the buzzwords sound adequate to describe Friday’s FDA approval of Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exa-cel) ...
FiercePharma

As Vertex preps for exa-cel launch, exec touts 'very large' opportunity but tempers early expectations

After a long development road, the first CRISPR-based gene editing therapy recently cleared a FDA advisory committee and appears to be nearing its U.S. launch. Vertex Pharmaceuticals, awaiting the ...