Fetal Gene Editing with Synthetic Nucleotides Cures Beta-Thalassemia in Mice Scientists at Yale University and Carnegie Mellon University have used gene editing to effectively cure the genetic blood disorder β-thalassemia, in mouse fetuses, in utero.  The approach, which harnesses peptide nucleic acid (PNA) technology pioneered at Carnegie Mellon’s Center for Nucleic Acids Science and Technology (CNAST), corrected the disease-causing β-globin …

Summary The company recently submitted a CTA for CTX-001 (the potential treatment for the blood disorder Beta-thalassemia). It’s the first application for the CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a potentially revolutionary platform to launch a vast number of individualized treatments for genetic diseases and cancers like sickle cell disease. With extensive resources/expertise, Vertex can help CRISPR to unlock substantial value …