<div>
  <p>
  </p>
  <ul>
    <li>
      <p>
        Vertex Pharmaceutical's and CRISPR Therapeutics's gene-editing therapy, CASGEVY (exa-cel), for treating transfusion-dependent beta-thalassemia has received FDA approval for patients aged 12 and older. This follows the FDA approval of gene editing therapy CASGEVY (exa-cel) for treating 
        <a href="https://sp-edge.com/updates/24785">
          sickle-cell disease
        </a>
         in December 2023.
      </p>
    </li>
    <li>
      <p>
        <b>Analyst QuickTake: </b>
        Casgevy&rsquo;s FDA approval becomes the world&rsquo;s first gene-editing treatment to enter the US market, marking a milestone in the human gene-editing industry. The approval follows Casgevy&rsquo;s market authorization in the UK in 
        <a href="https://sp-edge.com/updates/24093">
          November 2023
        </a>
        , in the Kingdom of Bahrain in 
        <a href="https://www.globenewswire.com/news-release/2023/12/02/2789644/0/en/CASGEVY-Gets-Bahrain-approval-for-treatment-marking-it-second-country-in-the-world.html">
          December 2023
        </a>
        , and in the Kingdom of Saudi Arabia 
        <a href="https://www.pharmaceutical-technology.com/news/vertexs-casgevy-saudi/">
          earlier this month
        </a>
        .&nbsp;&nbsp;
      </p>
    </li>
  </ul>
  <p>
  </p>
</div>


<div>
  <p>
    <a href="https://ir.crisprtx.com/news-releases/news-release-details/crispr-therapeutics-announces-us-food-and-drug-administration">
      Press release
    </a>
  </p>
  <p>
    <a href="https://www.forbes.com/sites/jamesfarrell/2024/01/16/fda-approves-new-crispr-gene-editing-treatment/?sh=4ef00c71b492">
      Forbes&nbsp;
    </a>
  </p>
  <p>
    <a href="https://www.pharmaceutical-technology.com/news/fda-approves-vertex-gene-therapy/?cf-view">
      Pharmaceutical Technology
    </a>
  </p>
  <p>
  </p>
</div>


Human Gene Editing

ANALYST_QUICK_TAKE

INCUMBENTS_PRESENT

<div>

<p>Vertex Pharmaceuticals and CRISPR Therapeutics have gained FDA approval for their gene-editing therapy, CASGEVY (exa-cel), aimed at treating transfusion-dependent beta-thalassemia in patients over 12 years. The approval follows a similar clearance given to CASGEVY (exa-cel) for treating <a href="https://sp-edge.com/updates/24785"> sickle-cell disease </a> back in December 2023.</p>

</div> <p><b>Analyst QuickTake: </b> Casgevy’s FDA approval becomes the world’s first gene-editing treatment to enter the US market, marking a milestone in the human gene-editing industry. The approval follows Casgevy’s market authorization in the UK in <a href="https://sp-edge.com/updates/24093"> November 2023 </a> , in the Kingdom of Bahrain in <a href="https://www.globenewswire.com/news-release/2023/12/02/2789644/0/en/CASGEVY-Gets-Bahrain-approval-for-treatment-marking-it-second-country-in-the-world.html"> December 2023 </a> , and in the Kingdom of Saudi Arabia <a href="https://www.pharmaceutical-technology.com/news/vertexs-casgevy-saudi/"> earlier this month </a> .&nbsp;&nbsp;</p>

Vertex-CRISPR beta-thalassemia drug receives FDA approval

PROMINENT_CATEGORY

<div>
<p>Editas Medicine, a Massachusetts-based startup, has agreed to permit Vertex Pharmaceuticals non-exclusively to use its Cas9 gene-editing technology. Editas will receive a total of USD 100 million from the deal, consisting of two payments of USD 50 million, plus annual license fees until 2034. Vertex will utilize the technology in the production of drugs for sickle cell disease and beta-thalassemia, including the newly approved drug CASGEVY. The deal will secure Editas Medicine's finances until 2026, despite payments due to The Broad Institute and Harvard College.</p>
</div>

Editas and Vertex collaborate to license Cas9 gene-editing technology

<div>
 
 <p>Vertex Pharmaceuticals and CRISPR Therapeutics secured FDA approval for their CASGEVY (exa-cel) gene-editing therapy, providing treatment for sickle-cell disease in patients aged 12 and up. The initial availability targets up to 16,000 patients in the US with the one-time therapy costing USD 2.2 million. Vertex will handle the global development, commercialization, and manufacturing of CASGEVY with CRISPR’s assistance, alongside a USD 200 million payment to CRISPR.</p>
 
</div> <p><b>Analyst QuickTake: </b> Casgevy’s FDA approval becomes the world’s first gene-editing treatment to enter the US market, marking a milestone in the human gene-editing industry. The approval follows Casgevy’s market authorization in the UK in <a href="https://sp-edge.com/updates/24093"> November 2023 </a> and in the Kingdom of Bahrain <a href="https://www.globenewswire.com/news-release/2023/12/02/2789644/0/en/CASGEVY-Gets-Bahrain-approval-for-treatment-marking-it-second-country-in-the-world.html"> early this month. </a> Despite successfully passing the FDA process, Casgevy is still expected to face certain speed breakers, such as pricing—one of the major obstacles in the market—and therapy implementation, like the collection of stem cells, ex-vivo manipulation, and treatment with chemotherapy to eradicate the blood system.&nbsp;&nbsp;&nbsp;</p>

Vertex-CRISPR’s sickle-cell drug becomes world’s first FDA-approved gene-editing drug

The gene editing therapy, exagamglogene autotemcel (exa-cel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, has received authorization from the UK's Medicines and Healthcare Products Regulatory Agency for the treatment of sickle cell disease and beta-thalassemia in patients aged 12 and older. The drug will be initially available for up to 2,000 patients in the UK. The price of the treatment has not been disclosed. <p><b>Analyst QuickTake:</b> Initiated as part of a collaboration between Vertex and CRISPR in 2015, Casgevy marks the first CRISPR-based gene editing treatment to receive market authorization for two blood disorders. The drug is also nearing its market entry in the US, with the FDA’s decision expected on December 8, 2023 (exa-cel for sickle cell disease patients) and March 30, 2024 (for beta-thalassemia patients). Just <a href="https://sp-edge.com/updates/23331"> last month </a> , exa-cel received positive remarks from the FDA’s advisory team indicating a potentially positive decision from the FDA.</p>

Vertex-CRISPR’s gene editing drug Casgevy gets world-first approval in UK

<div>
 <ul>
  <li><p>Vertex Pharmaceuticals and CRISPR Therapeutics's gene editing therapy, exagamglogene autotemcel (exa-cel), for the treatment of sickle cell disease in patients aged 12 and older, has successfully passed through the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee, reportedly the last step before the FDA's final decision. The FDA is expected to make a decision on the therapy by December 8.</p></li>
  <li><p>The therapy, which aims to restore normal red blood cell function and alleviate symptoms, has proved to be safe and effective in preventing painful episodes associated with the disease. However, there are concerns about potential long-term safety issues.</p></li>
  <li><p>Exa-cel is the first therapy developed by the duo, which entered into a partnership in October 2015. If approved by the FDA, it will be the first gene editing therapy available for SCD patients.</p></li>
 </ul> 
</div>
Summary: Vertex Pharmaceuticals and CRISPR Therapeutics have developed exagamglogene autotemcel (exa-cel), a gene editing therapy for sickle cell disease. It has passed the FDA's advisory committee and a decision is expected by December 8. The therapy aims to restore normal red blood cell function and alleviate symptoms, effectively preventing painful episodes. However, concerns about its long-term safety remain. If approved, it will be the first gene editing therapy for SCD patients.

Vertex-CRISPR sickle cell gene editing treatment exa-cel nears FDA approval

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All Updates

Vertex-CRISPR beta-thalassemia drug receives FDA approval

Editas and Vertex collaborate to license Cas9 gene-editing technology

Vertex-CRISPR’s sickle-cell drug becomes world’s first FDA-approved gene-editing drug

Vertex-CRISPR’s gene editing drug Casgevy gets world-first approval in UK

Vertex-CRISPR sickle cell gene editing treatment exa-cel nears FDA approval

Arbor Biotechnologies and Vertex Pharmaceuticals partner to develop genetic medicines

Arbor Biotechnologies and Vertex Pharmaceuticals partner to develop genetic medicines

Human Gene Editing

Vertex-CRISPR beta-thalassemia drug receives FDA approval

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