Sickle 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 misshapen or “sickled” blood cells.

Dec 8, 2023 · Vertex and CRISPR Therapeutics Announce US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Sickle Cell Disease – First-ever approval of a CRISPR-based gene-editing therapy in the U.S. –

Dec 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.

Dec 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). The therapy...

We work with organizations that help people with sickle cell disease (SCD) live active, full lives. We are committed to investigating and advancing approaches to treating SCD. Vertex seeks to identify opportunities where we can support, empower, learn from and engage with sickle cell warriors and ensure their voices are represented in all that ...

Jan 16, 2024 · All nine ATCs activated in the U.S. are able to offer CASGEVY to eligible patients with TDT and sickle cell disease (SCD). Additional ATCs will be activated in the coming weeks and a complete list of ATCs can be accessed at CASGEVY.com. About Transfusion-Dependent Beta Thalassemia (TDT) TDT is a serious, life-threatening genetic disease.

Nov 16, 2023 · - First regulatory authorization of a CRISPR-based gene-editing therapy in the world – - CASGEVY is indicated for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises who have the βS/βS, βS/β+ or βS/β0 genotype, for whom hematopoietic...

Dec 15, 2023 · The company proved the gene fix can be a permanent remedy for people who have the most severe sickle-cell symptoms. These individuals, numbering around 16,000 in the U.S., suffer recurring...

At Vertex, we invest in scientific innovation to create transformative medicines for people with serious diseases. We’re focused on discovering, developing and producing innovative medicines so people with serious diseases can lead better lives.

Jun 9, 2023 · Vertex and CRISPR Therapeutics’ dream of getting the first-ever CRISPR gene editing therapy approved now seems within reach as the candidate aced primary endpoints in a pair of phase 3 trials...