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Cilta-cel | November 01, 2021
Legend Biotech Corporation (Legend Biotech), a global, clinical-stage biotechnology company developing and manufacturing novel therapies, today announced that the U.S. Food and Drug Administration has extended the Prescription Drug User Fee Act (PDUFA) target date for ciltacabtagene autoleucel (cilta-cel) to February 28, 2022. Cilta-cel is a BCMA-directed chimeric antigen receptor T cell (CAR-T) therapy being investigated for the treatment of adults with relapsed and/or refractory multiple myelo...
Drug Target Review | January 15, 2020
Scientists have created the first lab-dish models of the cellular clock, where each ‘tick’ stimulates the formation of the vertebra, which uses stem cells derived from adult human tissue. Over 20 years ago, Olivier Pourquié’s lab at Harvard University, US discovered a cellular clock in chicken embryos where each ‘tick’ stimulates the formation of a structure called a somite that ultimately becomes a vertebra. Now, Pourquié has led one of two teams to c...
Labiotech | January 15, 2020
The US company bluebird bio has launched the first gene therapy for the blood disorder transfusion-dependent beta thalassemia in the EU, with a hospital in Germany becoming its first qualified treatment center. Bluebird bio’s gene therapy, branded as Zynteglo, was given conditional market approval by the EU in June 2019. It was the first gene therapy approved to treat people over 12 years of age with all but the most severe form of transfusion-dependent beta thalassemia, a condition that n...
Genetic Literacy Project | January 14, 2020
Researchers from Weill Cornell Medical College published a new study (“Anti-Phospho-Tau Gene Therapy for Chronic Traumatic Encephalopathy”) in Human Gene Therapy that shows the feasibility of using gene therapy to treat the progressive neurodegenerative disorder chronic traumatic encephalopathy (CTE). The study demonstrated the effectiveness of direct delivery of gene therapy into the brain of a mouse model of CTE....
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