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CRISPR Exa-cel

CRISPR Exa-cel
Date: 4/21/2023

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a powerful gene editing technology that allows researchers to precisely edit DNA sequences within cells. CRISPR works by using a specialized protein called Cas9, which can be programmed to target and cut specific DNA sequences. Once the DNA is cut, researchers can insert, remove, or replace genetic material as needed.

CRISPR gene editing has many potential applications in medicine, agriculture, and biotechnology. For example, it could be used to treat genetic diseases by correcting or removing the faulty genes responsible for those conditions. It could also be used to create crops that are resistant to pests or tolerant to drought, or to develop new therapies for cancer or other diseases.

CRISPR began human trials back in 2019. There have been several trials. Exa-cel (exagamglogene autotemcel) has been given fast track approval designation with the FDA due to its effectiveness in treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Here are the results of the 75 patient study.

31 Sickle Cell Disease patients (SCD) treated with Exa-cel(exagamglogene autotemcel) were free of disease symptoms at a long term follow up.

42 of 44 Transfusion-Dependent beta Thalassemia patients were treated with the exa-cel no longer needed transfusions and 2 had a 75-89% reduction in transfusion volumes.

See the article in The New Atlas 

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