<code id='034E7E6F97'></code><style id='034E7E6F97'></style>
    • <acronym id='034E7E6F97'></acronym>
      <center id='034E7E6F97'><center id='034E7E6F97'><tfoot id='034E7E6F97'></tfoot></center><abbr id='034E7E6F97'><dir id='034E7E6F97'><tfoot id='034E7E6F97'></tfoot><noframes id='034E7E6F97'>

    • <optgroup id='034E7E6F97'><strike id='034E7E6F97'><sup id='034E7E6F97'></sup></strike><code id='034E7E6F97'></code></optgroup>
        1. <b id='034E7E6F97'><label id='034E7E6F97'><select id='034E7E6F97'><dt id='034E7E6F97'><span id='034E7E6F97'></span></dt></select></label></b><u id='034E7E6F97'></u>
          <i id='034E7E6F97'><strike id='034E7E6F97'><tt id='034E7E6F97'><pre id='034E7E6F97'></pre></tt></strike></i>

          Home / Wikipedia / comprehensive

          comprehensive


          comprehensive

          author:focus    Page View:7884
          Christine Kao/STAT

          The Food and Drug Administration on Friday approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder.

          The new medicine, called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics. Its authorization is a scientific triumph for the technology that can efficiently and precisely repair DNA mutations — ushering in a new era of genetic medicines for inherited diseases.

          advertisement

          In a clinical trial, Casgevy was shown to eliminate recurrent episodes of debilitating pain caused by sickle cell, which afflicts approximately 100,000 people in the U.S., a vast majority of whom are Black. The therapy, whose scientific name is exa-cel, is described as a potential cure because the genetic fix enabled by CRISPR is designed to last a lifetime, although confirmation will require years of follow-up.

          Get unlimited access to award-winning journalism and exclusive events.

          Subscribe Log In