May 28, 2024

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Milestone: FDA accepts first marketing application for CRISPR gene-editing therapy

Milestone: FDA accepts first marketing application for CRISPR gene-editing therapy



 

Milestone: FDA accepts first marketing application for CRISPR gene-editing therapy.


On June 8, 2023, Vertex Pharmaceuticals and CRISPR Therapeutics announced that the U.S. Food and Drug Administration (FDA) accepted the CRISPR gene editing therapy exagamglogene autotemcel (exa-cel) for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta- Biologics License Application (BLA) for thalassemia (TDT) and priority review for the treatment of SCD.

It is reported that this is the first marketing application for CRISPR gene editing therapy accepted by FDA.

 

Milestone: FDA accepts first marketing application for CRISPR gene-editing therapy

 

Dr. Reshma Kewalramani , CEO and President of Vertex, said that he is very pleased that the FDA accepted exa-cel’s application and granted priority review status to its treatment of SCD.

Exa-cel is expected to become the first approved CRISPR gene editing therapy, and we will continue to work hard work to bring this transformative potential treatment to patients who are waiting.

 

Dr. Samarth Kulkarni , CEO of CRISPR , said that he is very pleased to see that the FDA has granted exa-cel the priority review status for the treatment of SCD . to patients in need.

 

On January 26, 2023, the European Medicines Agency (EMA) has also accepted the marketing application of exa-cel. This means that exa-cel is expected to be approved in Europe this year.

 

exa-cel is an autologous cell therapy based on CRISPR-Cas9 gene editing developed by CRISPR and Vertex for the treatment of sickle cell disease and β – thalassemia, which can help patients effectively get rid of blood transfusion and vascular occlusion crisis .

 

The clinical trial data of exa-cel therapy were previously published in the New England Journal of Medicine (NEJM) in December 2020 , titled : CRISPR – Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia .

 

Milestone: FDA accepts first marketing application for CRISPR gene-editing therapy

 


Therapeutic Principles and Clinical Trial Results

BCL11A is a transcription factor that represses γ-globin and fetal hemoglobin expression in erythroid cells.

Therefore, targeted inhibition of BCL11A could theoretically reactivate γ-globin expression for the treatment of thalassemia and sickle cell disease.

 

The research team obtained CD34+ hematopoietic stem and progenitor cells from healthy donors, and then introduced the CRISPR-Cas9 gene editing system specifically targeting the BCL11A enhancer by electroporation .

The detection results showed that about 80% of the alleles at this site were edited, and there was no sign of off-target editing.

 

Milestone: FDA accepts first marketing application for CRISPR gene-editing therapy

 

Then the research team reinfused autologous CD34+ cells that had been edited with CRISPR-Cas9 BCL11A enhancer to a patient with β-thalassemia and a patient with sickle cell disease .

 

Eighteen months after treatment, the levels of allelic editing in the bone marrow and blood of the two patients remained high, with an editing efficiency of more than 76% in the bone marrow and 60% in the blood. And the patient no longer depends on blood transfusion, and the proportion of cells expressing fetal hemoglobin HbF in circulating blood reaches 99%.

 

The founder of CRISPR Therapeutics is Emmanulle Charpentier, who won the Nobel Prize in Chemistry for CRISPR gene editing in 2020 .

 

 

 

 

 

 

References :

https://www.nejm.org/doi/10.1056/NEJMoa2031054

https://crisprtx.com/about-us/press-releases-and-presentations/fda-accepts-biologics-license-applications-for-exagamglogene-autotemcel-exa-cel-for-severe-sickle-cell-disease- and-transfusion-dependent-beta-thalassemia

Milestone: FDA accepts first marketing application for CRISPR gene-editing therapy

(source:internet, reference only)


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