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"ET-01" is a code name that refers to at least three distinct investigational compounds. One form of ET-01 is a topical formulation of botulinum toxin, Type A, which has been investigated for the reduction of lateral canthal lines, commonly known as Crow's Feet. Clinical trials for this application have been completed. A second distinct entity also designated as ET-01 involves autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells, which were under investigation for transplantation in subjects with transfusion-dependent β-thalassemia, though clinical trials for this gene and cell therapy approach have been terminated. A third distinct chemical entity referred to as ET-01 is an asymmetric trityl radical, studied for its redox properties and reactivity with biological oxidoreductants such as glutathione and superoxide anion radical.
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