Press Release - Vertex Pharmaceuticals Newsroom
WEBDec 8, 2023 · BOSTON & ZUG, Switzerland--(BUSINESS WIRE)--Dec. 8, 2023-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 genome-edited cell therapy, for the treatment of sickle ...
Vertex Pharmaceuticals | R&D Pipeline | Sickle Cell Disease
WEBSickle cell disease (SCD) is an inherited blood disorder that affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to …
Press Release - Vertex Pharmaceuticals Newsroom
WEBNov 16, 2023 · The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases.
FDA approves cure for sickle cell disease, the first treatment to …
WEBDec 8, 2023 · The therapy, called Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine to be approved in the United States that uses the gene-editing tool CRISPR, which won its...
FDA signs off on Vertex, CRISPR's gene therapy for sickle cell
WEBDec 8, 2023 · None of the buzzwords sound adequate to describe Friday’s FDA approval of Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exa-cel) to treat sickle cell disease (SCD).
I received the new gene-editing drug for sickle cell disease. It ...
WEBDec 4, 2023 · The package was from Vertex Pharmaceuticals, and it contained a consent form to participate in a clinical trial for a new gene-editing drug to treat sickle-cell disease.
Exagamglogene Autotemcel for Severe Sickle Cell Disease
WEBApr 24, 2024 · Exagamglogene autotemcel (exa-cel) is a nonviral, autologous cell therapy that is designed to reactivate fetal hemoglobin production by means of ex vivo clustered regularly interspaced...
News & Events - Vertex Pharmaceuticals
WEBJan 16, 2024 · The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases.
Two gene therapies for sickle cell approved by FDA | AP News
WEBDec 8, 2023 · The goal of the Vertex therapy, called Casgevy, is to help the body go back to producing a fetal form of hemoglobin that’s present at birth — it’s the adult form that’s defective in people with sickle cell disease.
FDA Approves First Gene Therapies to Treat Patients with Sickle Cell ...
WEBCasgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises.