Innovative Therapies
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Clinical News
FDA Grants RMAT Designation to CTX-001™ for the Treatment of Severe TDT β-Thalassaemia & SCD
CRISPR Therapeutics and Vertex Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX-001, an investigational, autologous, Gene Editing hematopoietic stem cell therapy, for the treatment of severe Transfusion-Dependent β-Thalassaemia (TDT) and Sickle…
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TIF Publications
Gene Therapy in β-Thalassaemia and Other Haemoglobin Disorders (2019)
An educational leaflet aimed at improving the awareness and comprehension of people with thalassaemia and other haemoglobinopathies worldwide about gene therapy.
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