The orphan drug designation for TCR² Therapeutics’ Gavo-cel will facilitate research into a treatment for cholangiocarcinoma.
TCR² Therapeutics, a clinical stage cell therapy company, announced that FDA granted orphan drug designation to Gavo-cel for the treatment of cholangiocarcinoma on Sept. 2, 2021. Cholangiocarcinoma is a rare and aggressive form of cancer that forms in the bile ducts.
Approximately 80,000 individuals in the United States have some form of solid tumor expressing mesothelin, including individuals with cholangiocarcinoma. Mesothelin is a cell-surface glycoprotein highly expressed in various types of solid tumors. Overexpression of mesothelin is associated with a poorer prognosis in some cancers due to its role in both malignant transformation and tumor aggressiveness.
Gavo-cel is a novel cell therapy that recently completed a phase I/II clinical trial in patients with treatment refractory mesothelin-expressing solid tumors. The therapy consists of autologous genetically engineered T cells expressing a single-domain antibody that recognizes human mesothelin fused to the CD3-epsilon subunit which, upon expression, is incorporated into the endogenous T cell receptor complex.
Source: TCR² Therapeutics
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