The FDA has started a priority review of Vertex Pharma and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel) in sickle cell disease (SCD), with a decision on approval due by 8th December.

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has continued ... and SCD who had been treated with CTX001 (exagamglogene autotemcel or exa-cel) for between around 2 and ...

The approval of the first CRISPR-based therapy in late 2023—CRISPR Therapeutics and Vertex Pharmaceuticals’ Casgevy (exagamglogene autotemcel or exa-cel) for sickle cell disease and β ...

CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that its Chief Operating Officer, Julianne ...

Vertex is expected to benefit from remaining ... disease and transfusion-dependent β-thalassemia gene therapy Casgevy (exagamglogene autotemcel) -- which it is partnered with CRISPR Therapeutics ...

The CRISPR-based gene therapy Casgevy (exagamglogene autotemcel ... The therapy was co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics. The institute said patients aged 12 and over ...

Clinical trials have shown that the one-time gene therapy, exagamglogene autotemcel (exa ... between NHS officials and the drug manufacturer, Vertex Pharmaceuticals. In clinical trials, all ...

In late 2023, the Medicines and Healthcare Products Regulatory Agency (MHRA) approved Vertex Pharmaceuticals’ Casgevy (exagamglogene autotemcel) for sickle cell disease (SCD), marking the world ...

Next review: More evidence on exagamglogene autotemcel is being collected. After this, NICE will decide whether to recommend it for use in the NHS and update the guidance. It will be available with ...