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Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell...
The Food and Drug Administration approved two genetic treatments for sickle cell disease, including one that uses gene-editing. The approvals offer hope for patients and signal a new...
The therapy, called Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine to be approved in the United States that uses the gene-editing tool CRISPR, which won its...
The US Food and Drug Administration on Friday approved two gene-based treatments for sickle cell disease, including the first therapy that uses the gene-editing technique CRISPR, opening a new...
The U.S. Food and Drug Administration approved two new gene therapies to treat sickle cell disease — one a gene-editing and the other a gene-addition approach. Nearly 100,000 people in the United States — and millions worldwide — have been diagnosed with this painful, life-threatening genetic blood disease.
On December 8, 2023, the US Food and Drug Administration (FDA) approved two autologous gene therapy products, Lyfgenia (lovotibeglogene autotemcel by bluebird bio) and Casgevy (exagamglogene autotemcel by Vertex Pharmaceuticals), for the treatment of individuals with sickle cell disease (SCD) ages 12 years and older with recurrent vaso ...
The Food and Drug Administration appears poised to approve the first medical therapy that uses gene-editing to treat a disease. A committee of independent FDA advisers Tuesday praised the...
The Food and Drug Administration approved the first genetic treatments for the brutal blood disorder on Friday. The announcement marks the first time that a medical treatment that uses gene editing has become available in the U.S. NPR health correspondent Rob Stein joins us. Rob, thanks for being with us. ROB STEIN, BYLINE: Oh, my pleasure, Scott.
The agency also approved another more traditional gene therapy for the disease as well: lovo-cel (brand name: Lyfgenia) from bluebird bio, giving sickle cell patients two powerful new ways...
Kendric Cromer, 12, the first commercial patient for Bluebird Bio’s gene therapy to cure his sickle cell disease, in the hospital as his bone marrow stem cells were being removed for gene...