CRISPR-Based es ya una realidad terapéutica...

 

The FDA set a target action date of December 8 for a decision on whether to approve Vertex and CRISPR Therapeutics’ CRISPR-Cas9 gene edited therapy for treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), but the UK has beaten it to the punch.

The UK Medicines and Healthcare products Regulatory Agency has conditionally authorized the therapy. “Today is a historic day in science and medicine: this authorization of CASGEVY in Great Britain is the first regulatory authorization of a CRISPR-based therapy in the world,” said Reshma Kewalramani, Chief Executive Officer and President of Vertex, in a statement.

Casgevy (exagamglogene autotemcel [exa-cel]) has been approved for patients aged 12 years and over with “SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not available.”

In a clinical trial for SCD, 28 out of 29 patients were free of severe pain crises for at least 12 months after treatment. For TDT, 39 out of 42 patients did not need a red blood cell transfusion for at least 12 months following treatment, with the other patients seeing a reduction of more than 70 percent in the need for red cell transfusions. The MHRA says it will monitor the safety of the treatment through real-word safety data and post-authorization safety studies to be conducted by Vertex and CRISPR Therapeutics.

Mas