Pharma News Intelligence December 13, 2023
Vertex Pharmaceuticals secured the first FDA approval for a CRISPR-based gene therapy with their sickle cell drug, Casgevy.
Last week marked a monumental regulatory decision for CRISPR-based gene editing technologies. The United States Food and Drug Administration (FDA) approved the first two cell-based gene therapies for people with sickle cell disease (SCD). Among these two approvals was the first FDA-approved CRISPR-based gene editing therapy, Casgevy, manufactured by Vertex Pharmaceuticals. Bluebird Bio secured the other SCD therapy approval for its drug Lyfgenia.
“Sickle cell disease is a rare, debilitating, and life-threatening blood disorder with significant unmet need, and we are excited to advance the field, especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based...
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