Orphan Drug Designation Given to New Mesothelioma CAR T-Cell Therapy

In early 2023, the world-class Hospital of the University of Pennsylvania will open a groundbreaking clinical trial for mesothelioma patients utilizing a next-generation immunotherapy with a novel delivery platform. The U.S. Food and Drug Administration cleared the path in September by granting orphan drug designation for SynKIR-110, a CAR T-cell therapy designed to target solid tumors expressing a specific protein typically found in mesothelioma cancer. By granting the orphan drug designation, the FDA can provide financial incentives such as tax credits for clinical trials and seven years of market exclusivity. It is designed to help find answers for treating rare diseases or disorders that affect fewer than 200,000 people in the U.S. A first-in-human clinical trial, officially known as STAR-101, will be assessing preliminary efficacy, tolerability and safety after showing impressive results – including complete remissions – in earlier murine (rodent) models. As part of the clinical trial, SynKIR-110 also will be used on patients with bile duct (cholangiocarcinoma) and ovarian cancer, but only those expressing the cell-surface protein mesothelin. SynKIR-110 is a product of Verismo Therapeutics, a Philadelphia-based biotechnology company that has built the KIR-CAR delivery platform, a modified killer-like receptor designed to improve efficacy and persistence against aggressive solid tumors. It will be the first product to use the KIR-CAR platform. “The...
Source: Asbestos and Mesothelioma News - Category: Environmental Health Authors: Source Type: news